Related references
Note: Only part of the references are listed.Enhanced genome editing to ameliorate a genetic metabolic liver disease through co-delivery of adeno-associated virus receptor
Shuming Yin et al.
SCIENCE CHINA-LIFE SCIENCES (2022)
Precise genomic deletions using paired prime editing
Junhong Choi et al.
NATURE BIOTECHNOLOGY (2022)
CRISPR-Cas12-based nucleic acids detection systems
Ross Ka-Kit Leung et al.
METHODS (2022)
Homology-based repair induced by CRISPR-Cas nucleases in mammalian embryo genome editing
Xiya Zhang et al.
PROTEIN & CELL (2022)
Low-density lipoprotein receptor-related protein 1 is a CROPs-associated receptor for Clostridioides difficile toxin B
Shengjie Guo et al.
SCIENCE CHINA-LIFE SCIENCES (2022)
Genome-wide CRISPR activation screen identifies candidate receptors for SARS-CoV-2 entry
Shiyou Zhu et al.
SCIENCE CHINA-LIFE SCIENCES (2022)
Rational designs of in vivo CRISPR-Cas delivery systems
Cong-Fei Xu et al.
ADVANCED DRUG DELIVERY REVIEWS (2021)
Pitfalls in SARS-CoV-2 PCR diagnostics
Kerstin Wernike et al.
TRANSBOUNDARY AND EMERGING DISEASES (2021)
An Ultralocalized Cas13a Assay Enables Universal and Nucleic Acid Amplification-Free Single-Molecule RNA Diagnostics
Tian Tian et al.
ACS NANO (2021)
High-Fidelity CRISPR/Cas13a trans-Cleavage-Triggered Rolling Circle Amplified DNAzyme for Visual Profiling of MicroRNA
Ting Zhou et al.
ANALYTICAL CHEMISTRY (2021)
Aptamer-Linked CRISPR/Cas12a-Based Immunoassay
Hui Li et al.
ANALYTICAL CHEMISTRY (2021)
Amplification-free detection of SARS-CoV-2 with CRISPR-Cas13a and mobile phone microscopy
Parinaz Fozouni et al.
CELL (2021)
Genome-wide Screens Identify Lineage- and Tumor-Specific Genes Modulating MHC-I- and MHC-II-Restricted Immunosurveillance of Human Lymphomas
Devin Dersh et al.
IMMUNITY (2021)
A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells
Giang N. Nguyen et al.
NATURE BIOTECHNOLOGY (2021)
CRISPR RNA-guided integrases for high-efficiency, multiplexed bacterial genome engineering
Phuc Leo H. Vo et al.
NATURE BIOTECHNOLOGY (2021)
Targeting herpes simplex virus with CRISPR-Cas9 cures herpetic stromal keratitis in mice
Di Yin et al.
NATURE BIOTECHNOLOGY (2021)
CRISPR technologies for precise epigenome editing
Muneaki Nakamura et al.
NATURE CELL BIOLOGY (2021)
Screening for functional circular RNAs using the CRISPR-Cas13 system
Siqi Li et al.
NATURE METHODS (2021)
Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease
Erica B. Esrick et al.
NEW ENGLAND JOURNAL OF MEDICINE (2021)
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
H. Frangoul et al.
NEW ENGLAND JOURNAL OF MEDICINE (2021)
NOP10 predicts lung cancer prognosis and its associated small nucleolar RNAs drive proliferation and migration
Chunhong Cui et al.
ONCOGENE (2021)
Aptamer-based CRISPR/Cas12a assay for the ultrasensitive detection of extracellular vesicle proteins
Huilan Li et al.
TALANTA (2021)
A smartphone-read ultrasensitive and quantitative saliva test for COVID-19
Bo Ning et al.
SCIENCE ADVANCES (2021)
TRIM26 is a critical host factor for HCV replication and contributes to host tropism
Yisha Liang et al.
SCIENCE ADVANCES (2021)
Appendage Regeneration in Vertebrates: What Makes This Possible?
Valentina Daponte et al.
CELLS (2021)
In vivo cytidine base editing of hepatocytes without detectable off-target mutations in RNA and DNA
Lukas Villiger et al.
NATURE BIOMEDICAL ENGINEERING (2021)
Lentiviral delivery of co-packaged Cas9 mRNA and a Vegfa-targeting guide RNA prevents wet age-related macular degeneration in mice
Sikai Ling et al.
NATURE BIOMEDICAL ENGINEERING (2021)
Cas12a-based electrochemiluminescence biosensor for target amplification-free DNA detection
Peng-Fei Liu et al.
BIOSENSORS & BIOELECTRONICS (2021)
Massively parallel assessment of human variants with base editor screens
Ruth E. Hanna et al.
CELL (2021)
In vivo CD8+ T cell CRISPR screening reveals control by Fli1 in infection and cancer
Zeyu Chen et al.
CELL (2021)
Functional interrogation of DNA damage response variants with base editing screens
Raquel Cuella-Martin et al.
CELL (2021)
Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice
Adam C. Wilkinson et al.
NATURE COMMUNICATIONS (2021)
Comprehensive deletion landscape of CRISPR-Cas9 identifies minimal RNA-guided DNA-binding modules
Arik Shams et al.
NATURE COMMUNICATIONS (2021)
Genome-wide programmable transcriptional memory by CRISPR-based epigenome editing
James K. Nunez et al.
CELL (2021)
CRISPR/Cas Technology in Pig-to-Human Xenotransplantation Research
Natalia Ryczek et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2021)
An Update on Gene Therapy for Inherited Retinal Dystrophy: Experience in Leber Congenital Amaurosis Clinical Trials
Wei Chiu et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2021)
Accurate and sensitive analysis of Staphylococcus aureus through CRISPR-Cas12a based recycling signal amplification cascades for early diagnosis of skin and soft tissue infections
JingJing Wei
JOURNAL OF MICROBIOLOGICAL METHODS (2021)
In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates
Kiran Musunuru et al.
NATURE (2021)
In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels
Tanja Rothgangl et al.
NATURE BIOTECHNOLOGY (2021)
High-efficiency prime editing with optimized, paired pegRNAs in plants
Qiupeng Lin et al.
NATURE BIOTECHNOLOGY (2021)
Genome-wide specificity of prime editors in plants
Shuai Jin et al.
NATURE BIOTECHNOLOGY (2021)
Eliminating base-editor-induced genome-wide and transcriptome-wide off-target mutations
Lijie Wang et al.
NATURE CELL BIOLOGY (2021)
Single-nucleotide-level mapping of DNA regulatory elements that control fetal hemoglobin expression
Li Cheng et al.
NATURE GENETICS (2021)
Programmable RNA editing with compact CRISPR-Cas13 systems from uncultivated microbes
Chunlong Xu et al.
NATURE METHODS (2021)
Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1
Sander F. Garrelfs et al.
NEW ENGLAND JOURNAL OF MEDICINE (2021)
Noncanonical crRNAs derived from host transcripts enable multiplexable RNA detection by Cas9
Chunlei Jiao et al.
SCIENCE (2021)
Indiscriminate ssDNA cleavage activity of CRISPR-Cas12a induces no detectable off-target effects in mouse embryos
Yu Wei et al.
PROTEIN & CELL (2021)
CRISPR-Based COVID-19 Testing: Toward Next-Generation Point-of-Care Diagnostics
Uyanga Ganbaatar et al.
FRONTIERS IN CELLULAR AND INFECTION MICROBIOLOGY (2021)
Application of Animal Models in Cancer Research: Recent Progress and Future Prospects
Zhitao Li et al.
CANCER MANAGEMENT AND RESEARCH (2021)
Discrimination of single-point mutations in unamplified genomic DNA via Cas9 immobilized on a graphene field-effect transistor
Sarah Balderston et al.
NATURE BIOMEDICAL ENGINEERING (2021)
Amplification-free RNA detection with CRISPR-Cas13
Hajime Shinoda et al.
COMMUNICATIONS BIOLOGY (2021)
Efficient embryonic homozygous gene conversion via RAD51-enhanced interhomolog repair
Jonathan J. Wilde et al.
CELL (2021)
Enhancing prime editing by Csy4-mediated processing of pegRNA
Yao Liu et al.
CELL RESEARCH (2021)
Droplet Cas12a Assay Enables DNA Quantification from Unamplified Samples at the Single-Molecule Level
Huahua Yue et al.
NANO LETTERS (2021)
Base editing of haematopoietic stem cells rescues sickle cell disease in mice
Gregory A. Newby et al.
NATURE (2021)
Genome-wide interrogation of gene functions through base editor screens empowered by barcoded sgRNAs
Ping Xu et al.
NATURE BIOTECHNOLOGY (2021)
Detect-seq reveals out-of-protospacer editing and target-strand editing by cytosine base editors
Zhixin Lei et al.
NATURE METHODS (2021)
CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis
Julian D. Gillmore et al.
NEW ENGLAND JOURNAL OF MEDICINE (2021)
Identification of pathways modulating vemurafenib resistance in melanoma cells via a genome-wide CRISPR/Cas9 screen
Corinna Jie Hui Goh et al.
G3-GENES GENOMES GENETICS (2021)
A CRISPR-Cas autocatalysis-driven feedback amplification network for supersensitive DNA diagnostics
Kai Shi et al.
SCIENCE ADVANCES (2021)
Adenine base editing in an adult mouse model of tyrosinaemia
Chun-Qing Song et al.
NATURE BIOMEDICAL ENGINEERING (2020)
A heterodimer of evolved designer-recombinases precisely excises a human genomic DNA locus
Felix Lansing et al.
NUCLEIC ACIDS RESEARCH (2020)
Viable pigs after simultaneous inactivation of porcine MHC class I and three xenoreactive antigen genes GGTA1, CMAH and B4GALNT2
Konrad Fischer et al.
XENOTRANSPLANTATION (2020)
Clustered Regularly Interspaced Short Palindromic Repeats/Cas9-Mediated Lateral Flow Nucleic Acid Assay
Xusheng Wang et al.
ACS NANO (2020)
Rapid and sensitive exosome detection with CRISPR/Cas12a
Xianxian Zhao et al.
ANALYTICAL AND BIOANALYTICAL CHEMISTRY (2020)
Rolling Circular Amplification (RCA)-Assisted CRISPR/Cas9 Cleavage (RACE) for Highly Specific Detection of Multiple Extracellular Vesicle MicroRNAs
Ruixuan Wang et al.
ANALYTICAL CHEMISTRY (2020)
RCasFISH: CRISPR/dCas9-Mediated in Situ Imaging of mRNA Transcripts in Fixed Cells and Tissues
Meng Wang et al.
ANALYTICAL CHEMISTRY (2020)
Reactivation of γ-globin expression through Cas9 or base editor to treat β-hemoglobinopathies
Liren Wang et al.
CELL RESEARCH (2020)
Detection of 2019 novel coronavirus (2019-nCoV) by real-time RT-PCR
Victor M. Corman et al.
EUROSURVEILLANCE (2020)
A genome-wide long noncoding RNA CRISPRi screen identifies PRANCR as a novel regulator of epidermal homeostasis
Pengfei Cai et al.
GENOME RESEARCH (2020)
Functional DNA Regulated CRISPR-Cas12a Sensors for Point-of-Care Diagnostics of Non-Nucleic-Acid Targets
Ying Xiong et al.
JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2020)
Immune Responses to Viral Gene Therapy Vectors
Jamie L. Shirley et al.
MOLECULAR THERAPY (2020)
Clades of huge phages from across Earth's ecosystems
Basem Al-Shayeb et al.
NATURE (2020)
Evaluation and minimization of Cas9-independent off-target DNA editing by cytosine base editors
Jordan L. Doman et al.
NATURE BIOTECHNOLOGY (2020)
Fitness effects of CRISPR/Cas9-targeting of long noncoding RNA genes
Max A. Horlbeck et al.
NATURE BIOTECHNOLOGY (2020)
Reply to: Fitness effects of CRISPR/Cas9-targeting of long noncoding RNA genes
Ying Liu et al.
NATURE BIOTECHNOLOGY (2020)
Continuous evolution of SpCas9 variants compatible with non-G PAMs
Shannon M. Miller et al.
NATURE BIOTECHNOLOGY (2020)
Evolutionary classification of CRISPR-Cas systems: a burst of class 2 and derived variants
Kira S. Makarova et al.
NATURE REVIEWS MICROBIOLOGY (2020)
Generation ofGGTA1-/-β2M-/-CIITA-/-Pigs Using CRISPR/Cas9 Technology to Alleviate Xenogeneic Immune Reactions
Rui Fu et al.
TRANSPLANTATION (2020)
CRISPR/Cas12a-Mediated Interfacial Cleaving of Hairpin DNA Reporter for Electrochemical Nucleic Acid Sensing
Decai Zhang et al.
ACS SENSORS (2020)
CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors
Dan Wang et al.
CELL (2020)
RNA-guided retargeting of Sleeping Beauty transposition in human cells
Adrian Kovac et al.
ELIFE (2020)
Stability issues of RT-PCR testing of SARS-CoV-2 for hospitalized patients clinically diagnosed with COVID-19
Yafang Li et al.
JOURNAL OF MEDICAL VIROLOGY (2020)
False negative of RT-PCR and prolonged nucleic acid conversion in COVID-19: Rather than recurrence
Ai Tang Xiao et al.
JOURNAL OF MEDICAL VIROLOGY (2020)
Otoferlin gene editing in sheep via CRISPR-assisted ssODN-mediated Homology Directed Repair
A. Menchaca et al.
SCIENTIFIC REPORTS (2020)
CRISPR/Cas9-mediated metabolic pathway reprogramming in a novel humanized rat model ameliorates primary hyperoxaluria type 1
Rui Zheng et al.
KIDNEY INTERNATIONAL (2020)
Highly Parallel Profiling of Cas9 Variant Specificity
Jonathan L. Schmid-Burgk et al.
MOLECULAR CELL (2020)
Amelioration of an Inherited Metabolic Liver Disease through Creation of a De Novo Start Codon by Cytidine Base Editing
Lei Yang et al.
MOLECULAR THERAPY (2020)
Applying CRISPR-Cas12a as a Signal Amplifier to Construct Biosensors for Non-DNA Targets in Ultralow Concentrations
Junjie Li et al.
ACS SENSORS (2020)
Sequence-Specific Recognition of HIV-1 DNA with Solid-State CRISPR-Cas12a-Assisted Nanopores (SCAN)
Reza Nouri et al.
ACS SENSORS (2020)
CRISPR screen in regulatory T cells reveals modulators of Foxp3
Jessica T. Cortez et al.
NATURE (2020)
Massively multiplexed nucleic acid detection with Cas13
Cheri M. Ackerman et al.
NATURE (2020)
Combinatorial single-cell CRISPR screens by direct guide RNA capture and targeted sequencing
Joseph M. Replogle et al.
NATURE BIOTECHNOLOGY (2020)
Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity
Michelle F. Richter et al.
NATURE BIOTECHNOLOGY (2020)
CRISPR-Cas12-based detection of SARS-CoV-2
James P. Broughton et al.
NATURE BIOTECHNOLOGY (2020)
Rapid detection of African swine fever virus using Cas12a-based portable paper diagnostics
Shuhan Lu et al.
CELL DISCOVERY (2020)
Therapeutic base editing of human hematopoietic stem cells
Jing Zeng et al.
NATURE MEDICINE (2020)
A rationally engineered cytosine base editor retains high on-target activity while reducing both DNA and RNA off-target effects
Erwei Zuo et al.
NATURE METHODS (2020)
Lipid nanoparticles for nucleic acid delivery: Current perspectives
Eleni Samaridou et al.
ADVANCED DRUG DELIVERY REVIEWS (2020)
Lipid nanoparticle technology for therapeutic gene regulation in the liver
Dominik Witzigmann et al.
ADVANCED DRUG DELIVERY REVIEWS (2020)
An ultrasensitive and specific point-of-care CRISPR/Cas12 based lateral flow biosensor for the rapid detection of nucleic acids
Omar Mukama et al.
BIOSENSORS & BIOELECTRONICS (2020)
Generation of Nonhuman Primate Model of Cone Dysfunction through In Situ MV-Mediated CNGB3 Ablation
Qiang Lin et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2020)
Allosteric Probe-Initiated Wash-Free Method for Sensitive Extracellular Vesicle Detection through Dual Cycle-Assisted CRISPR-Cas12a
Xianxian Zhao et al.
ACS SENSORS (2020)
Comparison and application of different immunoassay methods for the detection of SARS-CoV-2
Jingyun He et al.
JOURNAL OF MEDICAL VIROLOGY (2020)
Rationally Designed APOBEC3B Cytosine Base Editors with Improved Specificity
Shuai Jin et al.
MOLECULAR CELL (2020)
Current Status and Challenges of DNA Base Editing Tools
You Kyeong Jeong et al.
MOLECULAR THERAPY (2020)
Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors
Andrew V. Anzalone et al.
NATURE BIOTECHNOLOGY (2020)
Inactivation of ApoC3 by CRISPR/Cas9 Protects Against Atherosclerosis in Hamsters
Mengmeng Guo et al.
CIRCULATION RESEARCH (2020)
In vivo base editing restores sensory transduction and transiently improves auditory function in a mouse model of recessive deafness
Wei-Hsi Yeh et al.
SCIENCE TRANSLATIONAL MEDICINE (2020)
Application of CRISPR-Cas12a Enhanced Fluorescence Assay Coupled with Nucleic Acid Amplification for the Sensitive Detection of African Swine Fever Virus
Dagang Tao et al.
ACS SYNTHETIC BIOLOGY (2020)
Cas12a Base Editors Induce Efficient and Specific Editing with Low DNA Damage Response
Xiao Wang et al.
CELL REPORTS (2020)
Detection of SARS-CoV-2 with SHERLOCK One-Pot Testing
Julia Joung et al.
NEW ENGLAND JOURNAL OF MEDICINE (2020)
Unbiased investigation of specificities of prime editing systems in human cells
Do Yon Kim et al.
NUCLEIC ACIDS RESEARCH (2020)
Ultrasensitive CRISPR-based diagnostic for field-applicable detection of Plasmodium species in symptomatic and asymptomatic malaria
Rose A. Lee et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2020)
Enhancement of trans-cleavage activity of Cas12a with engineered crRNA enables amplified nucleic acid detection
Long T. Nguyen et al.
NATURE COMMUNICATIONS (2020)
Ultrasensitive and visual detection of SARS-CoV-2 using all-in-one dual CRISPR-Cas12a assay
Xiong Ding et al.
NATURE COMMUNICATIONS (2020)
CRISPR-Cas12a-Assisted Multicolor Biosensor for Semiquantitative Point-of-Use Testing of the Nopaline Synthase Terminator in Genetically Modified Crops by Unaided Eyes
Di Huang et al.
ACS SYNTHETIC BIOLOGY (2020)
PCSK9Variants in Familial Hypercholesterolemia: A Comprehensive Synopsis
Qianyun Guo et al.
FRONTIERS IN GENETICS (2020)
Clinical validation of a Cas13-based assay for the detection of SARS-CoV-2 RNA
Maturada Patchsung et al.
NATURE BIOMEDICAL ENGINEERING (2020)
Allele-Specific Chromosome Removal after Cas9 Cleavage in Human Embryos
Michael Zuccaro et al.
CELL (2020)
Genome-edited, donor-derived allogeneic anti-CD19 chimeric antigen receptor T cells in paediatric and adult B-cell acute lymphoblastic leukaemia: results of two phase 1 studies
Reuben Benjamin et al.
LANCET (2020)
Efficient, Rapid, and Sensitive Detection of Plant RNA Viruses With One-Pot RT-RPA-CRISPR/Cas12a Assay
Rashid Aman et al.
FRONTIERS IN MICROBIOLOGY (2020)
A CRISPR-Cas12a-based specific enhancer for more sensitive detection of SARS-CoV-2 infection
Weiren Huang et al.
EBIOMEDICINE (2020)
Improving the anti-solid tumor efficacy of CAR-T cells by inhibiting adenosine signaling pathway
Na Li et al.
ONCOIMMUNOLOGY (2020)
An ultrasensitive hybridization chain reaction-amplified CRISPR-Cas12a aptasensor for extracellular vesicle surface protein quantification
Shan Xing et al.
THERANOSTICS (2020)
An RNA-Guided Cas9 Nickase-Based Method for Universal Isothermal DNA Amplification
Ting Wang et al.
ANGEWANDTE CHEMIE-INTERNATIONAL EDITION (2019)
Evaluating and Enhancing Target Specificity of Gene-Editing Nucleases and Deaminases
Daesik Kim et al.
ANNUAL REVIEW OF BIOCHEMISTRY, VOL 88 (2019)
Disruption of CTLA-4 expression on peripheral blood CD8+T cell enhances anti-tumor efficacy in bladder cancer
Wei Zhang et al.
CANCER CHEMOTHERAPY AND PHARMACOLOGY (2019)
Non-viral Delivery of Zinc Finger Nuclease mRNA Enables Highly Efficient In Vivo Genome Editing of Multiple Therapeutic Gene Targets
Anthony Conway et al.
MOLECULAR THERAPY (2019)
Precise RNA editing by recruiting endogenous ADARs with antisense oligonucleotides
Tobias Merkle et al.
NATURE BIOTECHNOLOGY (2019)
Highly efficient therapeutic gene editing of human hematopoietic stem cells
Yuxuan Wu et al.
NATURE MEDICINE (2019)
Identification of preexisting adaptive immunity to Cas9 proteins in humans
Carsten T. Charlesworth et al.
NATURE MEDICINE (2019)
Adeno-associated virus vector as a platform for gene therapy delivery
Dan Wang et al.
NATURE REVIEWS DRUG DISCOVERY (2019)
Cytosine base editor generates substantial off-target single-nucleotide variants in mouse embryos
Erwei Zuo et al.
SCIENCE (2019)
Cytosine, but not adenine, base editors induce genome-wide off-target mutations in rice
Shuai Jin et al.
SCIENCE (2019)
The desirable donor pig to eliminate all xenoreactive antigens
Joseph Ladowski et al.
XENOTRANSPLANTATION (2019)
Detection of unamplified target genes via CRISPR-Cas9 immobilized on a graphene field-effect transistor
Reza Hajian et al.
NATURE BIOMEDICAL ENGINEERING (2019)
CRISPR/Cas9-mediated in vivo gene targeting corrects hemostasis in newborn and adult factor IX-knockout mice
Lili Wang et al.
BLOOD (2019)
Human Neonatal Fc Receptor Is the Cellular Uncoating Receptor for Enterovirus B
Xin Zhao et al.
CELL (2019)
Highly Efficient and Marker-free Genome Editing of Human Pluripotent Stem Cells by CRISPR-Cas9 RNP and AAV6 Donor-Mediated Homologous Recombination
Renata M. Martin et al.
CELL STEM CELL (2019)
Transcriptome-wide off-target RNA editing induced by CRISPR-guided DNA base editors
Julian Grunewald et al.
NATURE (2019)
Increasing the specificity of CRISPR systems with engineered RNA secondary structures
D. Dewran Kocak et al.
NATURE BIOTECHNOLOGY (2019)
To BE or not to BE, that is the question
Jia Chen et al.
NATURE BIOTECHNOLOGY (2019)
Multiplexed detection of proteins, transcriptomes, clonotypes and CRISPR perturbations in single cells
Eleni P. Mimitou et al.
NATURE METHODS (2019)
The next generation of CRISPR-Cas technologies and applications
Adrian Pickar-Oliver et al.
NATURE REVIEWS MOLECULAR CELL BIOLOGY (2019)
HBB-deficient Macaca fascicularis monkey presents with human -thalassemia
Yan Huang et al.
PROTEIN & CELL (2019)
In vivo genome editing rescues photoreceptor degeneration via a Cas9/RecA-mediated homology-directed repair pathway
Yuan Cai et al.
SCIENCE ADVANCES (2019)
Off-target RNA mutation induced by DNA base editing and its elimination by mutagenesis
Changyang Zhou et al.
NATURE (2019)
Transposon-encoded CRISPR-Cas systems direct RNA-guided DNA integration
Sanne E. Klompe et al.
NATURE (2019)
Atypical behaviour and connectivity in SHANK3-mutant macaques
Yang Zhou et al.
NATURE (2019)
Continuous evolution of base editors with expanded target compatibility and improved activity
B. W. Thuronyi et al.
NATURE BIOTECHNOLOGY (2019)
Programmable RNA editing by recruiting endogenous ADAR using engineered RNAs
Liang Qu et al.
NATURE BIOTECHNOLOGY (2019)
Allele-specific gene editing prevents deafness in a model of dominant progressive hearing loss
Bence Gyorgy et al.
NATURE MEDICINE (2019)
RNA-guided DNA insertion with CRISPR-associated transposases
Jonathan Strecker et al.
SCIENCE (2019)
A cytosine deaminase for programmable single-base RNA editing
Omar O. Abudayyeh et al.
SCIENCE (2019)
CRISPR/Cas9 Screens Reveal Multiple Layers of B cell CD40 Regulation
Chang Jiang et al.
CELL REPORTS (2019)
Immune-orthogonal orthologues of AAV capsids and of Cas9 circumvent the immune response to the administration of gene therapy
Ana M. Moreno et al.
NATURE BIOMEDICAL ENGINEERING (2019)
CRISPR-Cas12a Coupled with Platinum Nanoreporter for Visual Quantification of SNVs on a Volumetric Bar-Chart Chip
Ning Shao et al.
ANALYTICAL CHEMISTRY (2019)
Systematic Immunotherapy Target Discovery Using Genome-Scale In Vivo CRISPR Screens in CD8 T Cells
Matthew B. Dong et al.
CELL (2019)
CRISPR DNA base editors with reduced RNA off-target and self-editing activities
Julian Grunewald et al.
NATURE BIOTECHNOLOGY (2019)
In vivo CRISPR screening in CD8 T cells with AAV-Sleeping Beauty hybrid vectors identifies membrane targets for improving immunotherapy for glioblastoma
Lupeng Ye et al.
NATURE BIOTECHNOLOGY (2019)
CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia
Lei Xu et al.
NEW ENGLAND JOURNAL OF MEDICINE (2019)
A CRISPR-Cas9 screen identifies essential CTCF anchor sites for estrogen receptor-driven breast cancer cell proliferation
Gozde Korkmaz et al.
NUCLEIC ACIDS RESEARCH (2019)
Exploring genetic interaction manifolds constructed from rich single-cell phenotypes
Thomas M. Norman et al.
SCIENCE (2019)
HOLMESv2: A CRISPR-Cas12b-Assisted Platform for Nucleic Acid Detection and DNA Methylation Quantitation
Linxian Li et al.
ACS SYNTHETIC BIOLOGY (2019)
CRISPR-Cas9 Gene Editing in Lizards through Microinjection of Unfertilized Oocytes
Ashley M. Rasys et al.
CELL REPORTS (2019)
Exploring the Trans-Cleavage Activity of CRISPR-Cas12a (cpf1) for the Development of a Universal Electrochemical Biosensor
Yifan Dai et al.
ANGEWANDTE CHEMIE-INTERNATIONAL EDITION (2019)
CRISPR/Cas9-mediated genome editing in nonhuman primates
Yu Kang et al.
DISEASE MODELS & MECHANISMS (2019)
Search-and-replace genome editing without double-strand breaks or donor DNA
Andrew V. Anzalone et al.
NATURE (2019)
New pathologic mechanisms in nucleotide repeat expansion disorders
C. M. Rodriguez et al.
NEUROBIOLOGY OF DISEASE (2019)
A CRISPR-Cas12a-derived biosensing platform for the highly sensitive detection of diverse small molecules
Mindong Liang et al.
NATURE COMMUNICATIONS (2019)
Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII
Hainan Chen et al.
SCIENTIFIC REPORTS (2019)
Cardiovascular disease models: A game changing paradigm in drug discovery and screening
Houman Savoji et al.
BIOMATERIALS (2019)
In vivo genome and base editing of a human PCSK9 knock-in hypercholesterolemic mouse model
Alba Carreras et al.
BMC BIOLOGY (2019)
Coupled Single-Cell CRISPR Screening and Epigenomic Profiling Reveals Causal Gene Regulatory Networks
Adam J. Rubin et al.
CELL (2019)
One Prime for All Editing
Li Yang et al.
CELL (2019)
High prevalence of Streptococcus pyogenes Cas9-reactive T cells within the adult human population
Dimitrios L. Wagner et al.
NATURE MEDICINE (2019)
Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10
Morgan L. Maeder et al.
NATURE MEDICINE (2019)
CRISPR/Cas Systems towards Next-Generation Biosensing
Yi Li et al.
TRENDS IN BIOTECHNOLOGY (2019)
Engineering of CRISPR-Cas12b for human genome editing
Jonathan Strecker et al.
NATURE COMMUNICATIONS (2019)
In vitro transcribed sgRNA causes cell death by inducing interferon release
Wei Mu et al.
PROTEIN & CELL (2019)
Cas12a-Based On-Site and Rapid Nucleic Acid Detection of African Swine Fever
Jing Bai et al.
FRONTIERS IN MICROBIOLOGY (2019)
Impact of Genes and Environment on Obesity and Cardiovascular Disease
Yoriko Heianza et al.
ENDOCRINOLOGY (2019)
Guide RNAs with embedded barcodes boost CRISPR-pooled screens
Shiyou Zhu et al.
GENOME BIOLOGY (2019)
Curing hemophilia A by NHEJ-mediated ectopic F8 insertion in the mouse
Jian-Ping Zhang et al.
GENOME BIOLOGY (2019)
PASTMUS: mapping functional elements at single amino acid resolution in human cells
Xinyi Zhang et al.
GENOME BIOLOGY (2019)
Continuous evolution of base editors with expanded target compatibility and improved activity
Benjamin W. Thuronyi et al.
NATURE BIOTECHNOLOGY (2019)
Reproducibility of CRISPR-Cas9 methods for generation of conditional mouse alleles: a multi-center evaluation
Channabasavaiah B. Gurumurthy et al.
GENOME BIOLOGY (2019)
Somatic cell nuclear transfer: failures, successes and the challenges ahead
Marta Czernik et al.
INTERNATIONAL JOURNAL OF DEVELOPMENTAL BIOLOGY (2019)
Cas9/sgRNA selective targeting of the P23H Rhodopsin mutant allele for treating retinitis pigmentosa by intravitreal AAV9.PHP.B-based delivery
Serena G. Giannelli et al.
HUMAN MOLECULAR GENETICS (2018)
Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents
Xue Gao et al.
NATURE (2018)
Orthologous CRISPR-Cas9 enzymes for combinatorial genetic screens
Fadi J. Najm et al.
NATURE BIOTECHNOLOGY (2018)
APOBEC3 induces mutations during repair of CRISPR-Cas9-generated DNA breaks
Liqun Lei et al.
NATURE STRUCTURAL & MOLECULAR BIOLOGY (2018)
APOBEC3 induces mutations during repair of CRISPR-Cas9-generated DNA breaks
Liqun Lei et al.
NATURE STRUCTURAL & MOLECULAR BIOLOGY (2018)
Detection of target DNA with a novel Cas9/sgRNAs-associated reverse PCR (CARP) technique
Beibei Zhang et al.
ANALYTICAL AND BIOANALYTICAL CHEMISTRY (2018)
CRISPR/Cas9-Mediated Knockout of DGK Improves Antitumor Activities of Human T Cells
In-Young Jung et al.
CANCER RESEARCH (2018)
Transcriptome Engineering with RNA-Targeting Type VI-D CRISPR Effectors
Silvana Konermann et al.
CELL (2018)
Custom-Made Oocytes to Clone Non-human Primates
Jose B. Cibelli et al.
CELL (2018)
A Huntingtin Knockin Pig Model Recapitulates Features of Selective Neurodegeneration in Huntington's Disease
Sen Yan et al.
CELL (2018)
Cloning of Macaque Monkeys by Somatic Cell Nuclear Transfer
Zhen Liu et al.
CELL (2018)
CRISPR-Cas12a has both cis- and trans-cleavage activities on single-stranded DNA
Shi-Yuan Li et al.
CELL RESEARCH (2018)
CRISPR RNAs trigger innate immune responses in human cells
Sojung Kim et al.
GENOME RESEARCH (2018)
Models and mechanisms of repeat expansion disorders: a worm's eye view
Paige Rudich et al.
JOURNAL OF GENETICS (2018)
An off-the-shelf fratricide-resistant CAR-T for the treatment of T cell hematologic malignancies
Matthew L. Cooper et al.
LEUKEMIA (2018)
Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy
Seuk-Min Ryu et al.
NATURE BIOTECHNOLOGY (2018)
PrePAIRing Cas9s for screening success
Zhuo Zhou et al.
NATURE BIOTECHNOLOGY (2018)
Base editing with a Cpf1-cytidine deaminase fusion
Xiaosa Li et al.
NATURE BIOTECHNOLOGY (2018)
Repair of double-strand breaks induced by CRISPR-Cas9 leads to large deletions and complex rearrangements
Michael Kosicki et al.
NATURE BIOTECHNOLOGY (2018)
Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol
Lili Wang et al.
NATURE BIOTECHNOLOGY (2018)
Dual gene activation and knockout screen reveals directional dependencies in genetic networks
Michael Boettcher et al.
NATURE BIOTECHNOLOGY (2018)
p53 inhibits CRISPR-Cas9 engineering in human pluripotent stem cells
Robert J. Ihry et al.
NATURE MEDICINE (2018)
CRISPR-Cas9 genome editing induces a p53-mediated DNA damage response
Emma Haapaniemi et al.
NATURE MEDICINE (2018)
Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia
A. A. Thompson et al.
NEW ENGLAND JOURNAL OF MEDICINE (2018)
MicroRNA-focused CRISPR-Cas9 library screen reveals fitness-associated miRNAs
Jessica S. Kurata et al.
RNA (2018)
Field-deployable viral diagnostics using CRISPR-Cas13
Cameron Myhrvold et al.
SCIENCE (2018)
CRISPR-Cas12a target binding unleashes indiscriminate single-stranded DNase activity
Janice S. Chen et al.
SCIENCE (2018)
Next-generation diagnostics with CRISPR
Daniel S. Chertow
SCIENCE (2018)
Multiplexed and portable nucleic acid detection platform with Cas13, Cas12a, and Csm6
Jonathan S. Gootenberg et al.
SCIENCE (2018)
Highly Effective and Low-Cost MicroRNA Detection with CRISPR-Cas9
Xin-Yuan Qiu et al.
ACS SYNTHETIC BIOLOGY (2018)
Ossabaw Pigs With a PCSK9 Gain-of-Function Mutation Develop Accelerated Coronary Atherosclerotic Lesions: A Novel Model for Preclinical Studies
Fang Yuan et al.
JOURNAL OF THE AMERICAN HEART ASSOCIATION (2018)
A Nonhuman Primate Transplantation Model to Evaluate Hematopoietic Stem Cell Gene Editing Strategies for beta-Hemoglobinopathies
Olivier Humbert et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2018)
Mapping a functional cancer genome atlas of tumor suppressors in mouse liver using AAV-CRISPR-mediated direct in vivo screening
Guangchuan Wang et al.
SCIENCE ADVANCES (2018)
CRISPR-Cas12a-assisted nucleic acid detection
Shi-Yuan Li et al.
CELL DISCOVERY (2018)
Functional Genetic Variants Revealed by Massively Parallel Precise Genome Editing
Eilon Sharon et al.
CELL (2018)
Direct Visualization of Single-Nucleotide Variation in mtDNA Using a CRISPR/Cas9-Mediated Proximity Ligation Assay
Kaixiang Zhang et al.
JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2018)
Large deletions induced by Cas9 cleavage
Fatwa Adikusuma et al.
NATURE (2018)
SIRT6 deficiency results in developmental retardation cynomolgus monkeys
Weiqi Zhang et al.
NATURE (2018)
Evolved Cas9 variants with broad PAM compatibility and high DNA specificity
Johnny H. Hu et al.
NATURE (2018)
Efficient base editing in methylated regions with a human APOBEC3A-Cas9 fusion
Xiao Wang et al.
NATURE BIOTECHNOLOGY (2018)
Engineered CRISPR-Cas9 nuclease with expanded targeting space
Hiroshi Nishimasu et al.
SCIENCE (2018)
Directed evolution of CRISPR-Cas9 to increase its specificity
Jungjoon K. Lee et al.
NATURE COMMUNICATIONS (2018)
CRISPR-typing PCR (ctPCR), a new Cas9-based DNA detection method
Qiao Wang et al.
SCIENTIFIC REPORTS (2018)
A severe atherosclerosis mouse model on the resistant NOD background
Xugang Wang et al.
DISEASE MODELS & MECHANISMS (2018)
Genome-wide screening for functional long noncocing RNAs in human cells by Cas9 targeting of splice sites
Ying Liu et al.
NATURE BIOTECHNOLOGY (2018)
Treatment of a metabolic liver disease by in vivo genome base editing in adult mice
Lukas Villiger et al.
NATURE MEDICINE (2018)
Programmed DNA destruction by miniature CRISPR-Cas14 enzymes
Lucas B. Harrington et al.
SCIENCE (2018)
CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type I
Nerea Zabaleta et al.
NATURE COMMUNICATIONS (2018)
A CRISPR-Cas9-triggered strand displacement amplification method for ultrasensitive DNA detection
Wenhua Zhou et al.
NATURE COMMUNICATIONS (2018)
Gene Editing of HIV-1 Co-receptors to Prevent and/or Cure Virus Infection
Alexander G. Allen et al.
FRONTIERS IN MICROBIOLOGY (2018)
Allele-Specific CRISPR-Cas9 Genome Editing of the Single-Base P23H Mutation for Rhodopsin-Associated Dominant Retinitis Pigmentosa
Pingjuan Li et al.
CRISPR JOURNAL (2018)
CRISPR-Cas9-mediated multiplex gene editing in CAR-T cells
Xiaojuan Liu et al.
CELL RESEARCH (2017)
Multiplex Genome Editing to Generate Universal CAR T Cells Resistant to PD1 Inhibition
Jiangtao Ren et al.
CLINICAL CANCER RESEARCH (2017)
CRISPRi-based genome-scale identification of functional long noncoding RNA loci in human cells
S. John Liu et al.
SCIENCE (2017)
Paired Design of dCas9 as a Systematic Platform for the Detection of Featured Nucleic Acid Sequences in Pathogenic Strains
Yihao Zhang et al.
ACS SYNTHETIC BIOLOGY (2017)
Cre-dependent Cas9-expressing pigs enable efficient in vivo genome editing
Kepin Wang et al.
GENOME RESEARCH (2017)
APOBEC: From mutator to editor
Bei Yang et al.
JOURNAL OF GENETICS AND GENOMICS (2017)
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
Stephen Russell et al.
LANCET (2017)
CRISPR-Mediated Base Editing Enables Efficient Disruption of Eukaryotic Genes through Induction of STOP Codons
Pierre Billon et al.
MOLECULAR CELL (2017)
CRISPR/Cas9-Mediated Genome Editing as a Therapeutic Approach for Leber Congenital Amaurosis 10
Guo-Xiang Ruan et al.
MOLECULAR THERAPY (2017)
Discovery of stimulation-responsive immune enhancers with CRISPR activation
Dimitre R. Simeonov et al.
NATURE (2017)
Genome-scale activation screen identifies a lncRNA locus regulating a gene neighbourhood
Julia Joung et al.
NATURE (2017)
Enhanced proofreading governs CRISPR-Cas9 targeting accuracy
Janice S. Chen et al.
NATURE (2017)
RNA targeting with CRISPR-Cas13
Omar O. Abudayyeh et al.
NATURE (2017)
CMTM6 maintains the expression of PD-L1 and regulates anti-tumour immunity
Marian L. Burr et al.
NATURE (2017)
Programmable base editing of A.T to G.C in genomic DNA without DNA cleavage
Nicole M. Gaudelli et al.
NATURE (2017)
CRISPR-Cas9 epigenome editing enables high-throughput screening for functional regulatory elements in the human genome
Tyler S. Klann et al.
NATURE BIOTECHNOLOGY (2017)
Synergistic drug combinations for cancer identified in a CRISPR screen for pairwise genetic interactions
Kyuho Han et al.
NATURE BIOTECHNOLOGY (2017)
Increasing the genome-targeting scope and precision of base editing with engineered Cas9-cytidine deaminase fusions
Y. Bill Kim et al.
NATURE BIOTECHNOLOGY (2017)
Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing
Hao Yin et al.
NATURE BIOTECHNOLOGY (2017)
A tiling-deletion-based genetic screen for cis-regulatory element identification in mammalian cells
Yarui Diao et al.
NATURE METHODS (2017)
Combinatorial CRISPR-Cas9 screens for de novo mapping of genetic interactions
John Paul Shen et al.
NATURE METHODS (2017)
CIRCLE-seq: a highly sensitive in vitro screen for genome-wide CRISPR Cas9 nuclease off-targets
Shengdar Q. Tsai et al.
NATURE METHODS (2017)
AAV-mediated direct in vivo CRISPR screen identifies functional suppressors in glioblastoma
Ryan D. Chow et al.
NATURE NEUROSCIENCE (2017)
Delivery technologies for genome editing
Hao Yin et al.
NATURE REVIEWS DRUG DISCOVERY (2017)
Comparative analysis of chimeric ZFP-, TALE- and Cas9-piggyBac transposases for integration into a single locus in human cells
Wentian Luo et al.
NUCLEIC ACIDS RESEARCH (2017)
RNA editing with CRISPR-Cas13
David B. T. Cox et al.
SCIENCE (2017)
Nucleic acid detection with CRISPR-Cas13a/C2c2
Jonathan S. Gootenberg et al.
SCIENCE (2017)
Inactivation of porcine endogenous retrovirus in pigs using CRISPR-Cas9
Dong Niu et al.
SCIENCE (2017)
Rett syndrome modeling goes simian
Gaia Novarino
Science Translational Medicine (2017)
Improving the DNA specificity and applicability of base editing through protein engineering and protein delivery
Holly A. Rees et al.
NATURE COMMUNICATIONS (2017)
CRISPR/Cas9-mediated PD-1 disruption enhances anti-tumor efficacy of human chimeric antigen receptor T cells
Levi J. Rupp et al.
SCIENTIFIC REPORTS (2017)
Advances in the delivery of RNA therapeutics: from concept to clinical reality
James C. Kaczmarek et al.
GENOME MEDICINE (2017)
CRISPR-Cas9 mediated LAG-3 disruption in CAR-T cells
Yongping Zhang et al.
FRONTIERS OF MEDICINE (2017)
Improved base excision repair inhibition and bacteriophage Mu Gam protein yields C:G-to-T: A base editors with higher efficiency and product purity
Alexis C. Komor et al.
SCIENCE ADVANCES (2017)
Clustered Regularly Interspaced Short Palindromic Repeats/Cas9 Triggered Isothermal Amplification for Site-Specific Nucleic Acid Detection
Mengqi Huang et al.
ANALYTICAL CHEMISTRY (2017)
34. Zinc Finger Nucleases Targeting the Glucocorticoid Receptor Allow IL-13 Zetakine Transgenic CTLs To Kill Glioblastoma Cells In Vivo in the Presence of Immunosuppressing Glucocorticoids
MOLECULAR THERAPY (2017)
Enhanced base editing by co-expression of free uracil DNA glycosylase inhibitor
Lijie Wang et al.
CELL RESEARCH (2017)
In Vivo Base Editing of PCSK9 (Proprotein Convertase Subtilisin/Kexin Type 9) as a Therapeutic Alternative to Genome Editing
Alexandra C. Chadwick et al.
ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY (2017)
Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9
Ranjan Batra et al.
CELL (2017)
CRISPR/Cas9-Mediated Scanning for Regulatory Elements Required for HPRT1 Expression via Thousands of Large, Programmed Genomic Deletions
Molly Gasperini et al.
AMERICAN JOURNAL OF HUMAN GENETICS (2017)
Modeling Rett Syndrome Using TALEN-Edited MECP2 Mutant Cynomolgus Monkeys
Yongchang Chen et al.
CELL (2017)
Gene therapy for neurological disorders
Chi-Jing Choong et al.
EXPERT OPINION ON BIOLOGICAL THERAPY (2016)
Efficient Generation of Gene-Modified Pigs Harboring Precise Orthologous Human Mutation via CRISPR/Cas9-Induced Homology-Directed Repair in Zygotes
Xiaoyang Zhou et al.
HUMAN MUTATION (2016)
A mouse model for adult cardiac-specific gene deletion with CRISPR/Cas9
Kelli J. Carroll et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2016)
Rationally engineered Cas9 nucleases with improved specificity
Ian M. Slaymaker et al.
SCIENCE (2016)
Circular RNAs: Identification, biogenesis and function
Karoline K. Ebbesen et al.
BIOCHIMICA ET BIOPHYSICA ACTA-GENE REGULATORY MECHANISMS (2016)
Rapid, Low-Cost Detection of Zika Virus Using Programmable Biomolecular Components
Keith Pardee et al.
CELL (2016)
PAM-Dependent Target DNA Recognition and Cleavage by C2c1 CRISPR-Cas Endonuclease
Hui Yang et al.
CELL (2016)
Dissecting Immune Circuits by Linking CRISPR-Pooled Screens with Single-Cell RNA-Seq
Diego Adhemar Jaitin et al.
CELL (2016)
Perturb-Seq: Dissecting Molecular Circuits with Scalable Single-Cell RNA Profiling of Pooled Genetic Screens
Atray Dixit et al.
CELL (2016)
A Multiplexed Single-Cell CRISPR Screening Platform Enables Systematic Dissection of the Unfolded Protein Response
Britt Adamson et al.
CELL (2016)
Editing DNA Methylation in the Mammalian Genome
X. Shawn Liu et al.
CELL (2016)
Generation of a Nonhuman Primate Model of Severe Combined Immunodeficiency Using Highly Efficient Genome Editing
Kenya Sato et al.
CELL STEM CELL (2016)
Cre Recombinase and Other Tyrosine Recombinases
Gretchen Meinke et al.
CHEMICAL REVIEWS (2016)
DNA Transposition at Work
Alison B. Hickman et al.
CHEMICAL REVIEWS (2016)
CRISPR/Cas9-mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse
Yuting Guan et al.
EMBO MOLECULAR MEDICINE (2016)
A new class of temporarily phenotypic enhancers identified by CRISPR/Cas9-mediated genetic screening
Yarui Diao et al.
GENOME RESEARCH (2016)
Genome editing in nonhuman primates: approach to generating human disease models
Y. Chen et al.
JOURNAL OF INTERNAL MEDICINE (2016)
Potent and Targeted Activation of Latent HIV-1 Using the CRISPR/dCas9 Activator Complex
Sheena M. Saayman et al.
MOLECULAR THERAPY (2016)
CRISPR Repair Reveals Causative Mutation in a Preclinical Model of Retinitis Pigmentosa
Wen-Hsuan Wu et al.
MOLECULAR THERAPY (2016)
In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa
Benjamin Bakondi et al.
MOLECULAR THERAPY (2016)
Specific Reactivation of Latent HIV-1 by dCas9-SunTag-VP64-mediated Guide RNA Targeting the HIV-1 Promoter
Haiyan Ji et al.
MOLECULAR THERAPY (2016)
CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells
Daniel P. Dever et al.
NATURE (2016)
Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage
Alexis C. Komor et al.
NATURE (2016)
High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects
Benjamin P. Kleinstiver et al.
NATURE (2016)
Two distinct RNase activities of CRISPR-C2c2 enable guide-RNA processing and RNA detection
Alexandra East-Seletsky et al.
NATURE (2016)
Genome-scale deletion screening of human long non-coding RNAs using a paired-guide RNA CRISPR-Cas9 library
Shiyou Zhu et al.
NATURE BIOTECHNOLOGY (2016)
High-throughput mapping of regulatory DNA
Nisha Rajagopal et al.
NATURE BIOTECHNOLOGY (2016)
Genome-wide specificities of CRISPR-Cas Cpf1 nucleases in human cells
Benjamin P. Kleinstiver et al.
NATURE BIOTECHNOLOGY (2016)
Functional genetic screens for enhancer elements in the human genome using CRISPR-Cas9
Gozde Korkmaz et al.
NATURE BIOTECHNOLOGY (2016)
Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo
Hao Yin et al.
NATURE BIOTECHNOLOGY (2016)
A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition
Elizabeth A. Traxler et al.
NATURE MEDICINE (2016)
Targeted AIDID-mediated mutagenesis (TAM) enables efficient genomic diversification in mammalian cells
Yunqing Ma et al.
NATURE METHODS (2016)
Directed evolution using dCas9-targeted somatic hypermutation in mammalian cells
Gaelen T. Hess et al.
NATURE METHODS (2016)
Multiplexed barcoded CRISPR-Cas9 screening enabled by CombiGEM
Alan S. L. Wong et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2016)
Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems
Keiji Nishida et al.
SCIENCE (2016)
Systematic mapping of functional enhancer-promoter connections with CRISPR interference
Charles P. Fulco et al.
SCIENCE (2016)
C2c2 is a single-component programmable RNA-guided RNA-targeting CRISPR effector
Omar O. Abudayyeh et al.
SCIENCE (2016)
Silencing porcine genes significantly reduces human-anti-pig cytotoxicity profiles: an alternative to direct complement regulation
James R. Butler et al.
TRANSGENIC RESEARCH (2016)
The APOBEC Protein Family: United by Structure, Divergent in Function
Jason D. Salter et al.
TRENDS IN BIOCHEMICAL SCIENCES (2016)
Repair Pathway Choices and Consequences at the Double-Strand Break
Raphael Ceccaldi et al.
TRENDS IN CELL BIOLOGY (2016)
Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia
Francis P. Pankowicz et al.
NATURE COMMUNICATIONS (2016)
Multiplexed pancreatic genome engineering and cancer induction by transfection-based CRISPR/Cas9 delivery in mice
Roman Maresch et al.
NATURE COMMUNICATIONS (2016)
Brain tumor modeling using the CRISPR/Cas9 system: state of the art and view to the future
Xiao-Yuan Mao et al.
ONCOTARGET (2016)
Compact and highly active next-generation libraries for CRISPR-mediated gene repression and activation
Max A. Horlbeck et al.
ELIFE (2016)
Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts Through BCL11A Knockdown Using Targeted Endonucleases
Carmen F. Bjurstrom et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2016)
In vivo Editing of the Human Mutant Rhodopsin Gene by Electroporation of Plasmid-based CRISPR/Cas9 in the Mouse Retina
Maria Carmela Latella et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2016)
Genome-Wide CRISPR-Cas9 Screen Identifies MicroRNAs That Regulate Myeloid Leukemia Cell Growth
Jared Wallace et al.
PLOS ONE (2016)
Efficient generation of B2m-null pigs via injection of zygote with TALENs
Yong Wang et al.
SCIENTIFIC REPORTS (2016)
Eukaryotic Mismatch Repair in Relation to DNA Replication
Thomas A. Kunkel et al.
ANNUAL REVIEW OF GENETICS, VOL 49 (2015)
Targeting hepatitis B virus cccDNA by CRISPR/Cas9 nuclease efficiently inhibits viral replication
Chunsheng Dong et al.
ANTIVIRAL RESEARCH (2015)
Genome-wide CRISPR Screen in a Mouse Model of Tumor Growth and Metastasis
Sidi Chen et al.
CELL (2015)
A Platform for Rapid Exploration of Aging and Diseases in a Naturally Short-Lived Vertebrate
Itamar Harel et al.
CELL (2015)
Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System
Bernd Zetsche et al.
CELL (2015)
High-throughput screens in mammalian cells using the CRISPR-Cas9 system
Jingyu Peng et al.
FEBS JOURNAL (2015)
Disruption of MeCP2 attenuates circadian rhythm in CRISPR/Cas9-based Rett syndrome model mouse
Yoshiki Tsuchiya et al.
GENES TO CELLS (2015)
Functional disruption of the dystrophin gene in rhesus monkey using CRISPR/Cas9
Yongchang Chen et al.
HUMAN MOLECULAR GENETICS (2015)
CRISPR-Cas9: A Revolutionary Tool for Cancer Modelling
Raul Torres-Ruiz et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2015)
Engineered CRISPR-Cas9 nucleases with altered PAM specificities
Benjamin P. Kleinstiver et al.
NATURE (2015)
BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis
Matthew C. Canver et al.
NATURE (2015)
In vivo genome editing using Staphylococcus aureus Cas9
F. Ann Ran et al.
NATURE (2015)
Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells
Ayal Hendel et al.
NATURE BIOTECHNOLOGY (2015)
Inducible in vivo genome editing with CRISPR-Cas9
Lukas E. Dow et al.
NATURE BIOTECHNOLOGY (2015)
Therapeutic genome editing: prospects and challenges
David Benjamin Turitz Cox et al.
NATURE MEDICINE (2015)
Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells
Daesik Kim et al.
NATURE METHODS (2015)
New treatment options for hearing loss
Ulrich Mueller et al.
NATURE REVIEWS DRUG DISCOVERY (2015)
High-throughput functional genomics using CRISPR-Cas9
Ophir Shalem et al.
NATURE REVIEWS GENETICS (2015)
Long-Term Effect of Gene Therapy on Leber's Congenital Amaurosis
J. W. B. Bainbridge et al.
NEW ENGLAND JOURNAL OF MEDICINE (2015)
Improvement and Decline in Vision with Gene Therapy in Childhood Blindness
Samuel G. Jacobson et al.
NEW ENGLAND JOURNAL OF MEDICINE (2015)
CASFISH: CRISPR/Cas9-mediated in situ labeling of genomic loci in fixed cells
Wulan Deng et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2015)
CRISPR/Cas9 somatic multiplex-mutagenesis for high-throughput functional cancer genomics in mice
Julia Weber et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2015)
Somatic CRISPR/Cas9-mediated tumour suppressor disruption enables versatile brain tumour modelling
Marc Zuckermann et al.
NATURE COMMUNICATIONS (2015)
CRISPR/Cas9 cleavage of viral DNA efficiently suppresses hepatitis B virus
Vyas Ramanan et al.
SCIENTIFIC REPORTS (2015)
Generation of gene-modified goats targeting MSTN and FGF5 via zygote injection of CRISPR/Cas9 system
Xiaolong Wang et al.
SCIENTIFIC REPORTS (2015)
Efficient generation of gene-modified pigs via injection of zygote with Cas9/sgRNA
Yong Wang et al.
SCIENTIFIC REPORTS (2015)
A Dual-Reporter System for Real-Time Monitoring and High-throughput CRISPR/Cas9 Library Screening of the Hepatitis C Virus
Qingpeng Ren et al.
SCIENTIFIC REPORTS (2015)
Both TALENs and CRISPR/Cas9 directly target the HBB IVS2-654 (C > T) mutation in β-thalassemia-derived iPSCs
Peng Xu et al.
SCIENTIFIC REPORTS (2015)
Generation of CRISPR/Cas9-mediated gene-targeted pigs via somatic cell nuclear transfer
Xiaoqing Zhou et al.
CELLULAR AND MOLECULAR LIFE SCIENCES (2015)
TALEN-mediated targeting of HPV oncogenes ameliorates HPV-related cervical malignancy
Zheng Hu et al.
JOURNAL OF CLINICAL INVESTIGATION (2015)
Promoter less gene targeting without nucleases ameliorates haemophilia B in mice
A. Barzel et al.
NATURE (2015)
In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9
Lukasz Swiech et al.
NATURE BIOTECHNOLOGY (2015)
Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo
John A. Zuris et al.
NATURE BIOTECHNOLOGY (2015)
GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases
Shengdar Q. Tsai et al.
NATURE BIOTECHNOLOGY (2015)
Genome-wide detection of DNA double-stranded breaks induced by engineered nucleases
Richard L. Frock et al.
NATURE BIOTECHNOLOGY (2015)
CRISPR-engineered mosaicism rapidly reveals that loss of Kcnj13 function in mice mimics human disease phenotypes
Hua Zhong et al.
SCIENTIFIC REPORTS (2015)
Precise gene deletion and replacement using the CRISPR/Cas9 system in human cells
Qiupeng Zheng et al.
BIOTECHNIQUES (2014)
Simultaneous zinc-finger nuclease editing of the HIV coreceptors ccr5 and cxcr4 protects CD4+ T cells from HIV-1 infection
Chuka A. Didigu et al.
BLOOD (2014)
CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling
Randall J. Platt et al.
CELL (2014)
Crystal Structure of Cas9 in Complex with Guide RNA and Target DNA
Hiroshi Nishimasu et al.
CELL (2014)
A Protein-Tagging System for Signal Amplification in Gene Expression and Fluorescence Imaging
Marvin E. Tanenbaum et al.
CELL (2014)
Generation of Gene-Modified Cynomolgus Monkey via Cas9/RNA-Mediated Gene Targeting in One-Cell Embryos
Yuyu Niu et al.
CELL (2014)
Generating rats with conditional alleles using CRISPR/Cas9
Yuanwu Ma et al.
CELL RESEARCH (2014)
Efficient Ablation of Genes in Human Hematopoietic Stem and Effector Cells using CRISPR/Cas9
Pankaj K. Mandal et al.
CELL STEM CELL (2014)
Low Incidence of Off-Target Mutations in Individual CRISPR-Cas9 and TALEN Targeted Human Stem Cell Clones Detected by Whole-Genome Sequencing
Adrian Veres et al.
CELL STEM CELL (2014)
PCSK9: A Key Modulator of Cardiovascular Health
Nabil G. Seidah et al.
CIRCULATION RESEARCH (2014)
Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing
Qiurong Ding et al.
CIRCULATION RESEARCH (2014)
Highly efficient CRISPR/Cas9-mediated knock-in in zebrafish by homology-independent DNA repair
Thomas O. Auer et al.
GENOME RESEARCH (2014)
Gene disruption by cell-penetrating peptide-mediated delivery of Cas9 protein and guide RNA
Suresh Ramakrishna et al.
GENOME RESEARCH (2014)
Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins
Sojung Kim et al.
GENOME RESEARCH (2014)
An Efficient Antiviral Strategy for Targeting Hepatitis B Virus Genome Using Transcription Activator-Like Effector Nucleases
Jieliang Chen et al.
MOLECULAR THERAPY (2014)
High-throughput screening of a CRISPR/Cas9 library for functional genomics in human cells
Yuexin Zhou et al.
NATURE (2014)
Structural basis of PAM-dependent target DNA recognition by the Cas9 endonuclease
Carolin Anders et al.
NATURE (2014)
Rapid modelling of cooperating genetic events in cancer through somatic genome editing
Francisco J. Sanchez-Rivera et al.
NATURE (2014)
CRISPR-mediated direct mutation of cancer genes in the mouse liver
Wen Xue et al.
NATURE (2014)
DNA interrogation by the CRISPR RNA-guided endonuclease Cas9
Samuel H. Sternberg et al.
NATURE (2014)
Improving CRISPR-Cas nuclease specificity using truncated guide RNAs
Yanfang Fu et al.
NATURE BIOTECHNOLOGY (2014)
Dimeric CRISPR RNA-guided Fokl nucleases for highly specific genome editing
Shengdar Q. Tsai et al.
NATURE BIOTECHNOLOGY (2014)
Fusion of catalytically inactive Cas9 to Fokl nuclease improves the specificity of genome modification
John P. Guilinger et al.
NATURE BIOTECHNOLOGY (2014)
Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype
Hao Yin et al.
NATURE BIOTECHNOLOGY (2014)
Efficient genome modification by CRISPR-Cas9 nickase with minimal off-target effects
Bin Shen et al.
NATURE METHODS (2014)
CRISPR/Cas-mediated genome editing in the rat via direct injection of one-cell embryos
Yanjiao Shao et al.
NATURE PROTOCOLS (2014)
Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV
Pablo Tebas et al.
NEW ENGLAND JOURNAL OF MEDICINE (2014)
Loss-of-Function Mutations in APOC3, Triglycerides, and Coronary Disease
Jacy Crosby et al.
NEW ENGLAND JOURNAL OF MEDICINE (2014)
ClinVar: public archive of relationships among sequence variation and human phenotype
Melissa J. Landrum et al.
NUCLEIC ACIDS RESEARCH (2014)
TALENs facilitate targeted genome editing in human cells with high specificity and low cytotoxicity
Claudio Mussolino et al.
NUCLEIC ACIDS RESEARCH (2014)
Natural History of Cone Disease in the Murine Model of Leber Congenital Amaurosis Due to CEP290 Mutation: Determining the Timing and Expectation of Therapy
Shannon E. Boye et al.
PLOS ONE (2014)
AAV-Mediated Delivery of Zinc Finger Nucleases Targeting Hepatitis B Virus Inhibits Active Replication
Nicholas D. Weber et al.
PLOS ONE (2014)
RNA-guided endonuclease provides a therapeutic strategy to cure latent herpesviridae infection
Jianbin Wang et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2014)
Genome-Scale CRISPR-Cas9 Knockout Screening in Human Cells
Ophir Shalem et al.
SCIENCE (2014)
Genetic Screens in Human Cells Using the CRISPR-Cas9 System
Tim Wang et al.
SCIENCE (2014)
Modernizing the Nonhomologous End-Joining Repertoire: Alternative and Classical NHEJ Share the Stage
Ludovic Deriano et al.
ANNUAL REVIEW OF GENETICS, VOL 47 (2013)
Toward eliminating HLA class I expression to generate universal cells from allogeneic donors
Hiroki Torikai et al.
BLOOD (2013)
One-Step Generation of Mice Carrying Mutations in Multiple Genes by CRISPR/Cas-Mediated Genome Engineering
Haoyi Wang et al.
CELL (2013)
Repurposing CRISPR as an RNA-Guided Platform for Sequence-Specific Control of Gene Expression
Lei S. Qi et al.
CELL (2013)
One-Step Generation of Mice Carrying Reporter and Conditional Alleles by CRISPR/Cas-Mediated Genome Engineering
Hui Yang et al.
CELL (2013)
Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity
F. Ann Ran et al.
CELL (2013)
CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes
Luke A. Gilbert et al.
CELL (2013)
Genome editing with RNA-guided Cas9 nuclease in Zebrafish embryos
Nannan Chang et al.
CELL RESEARCH (2013)
Inactivation of Hepatitis B Virus Replication in Cultured Cells and In Vivo with Engineered Transcription Activator-Like Effector Nucleases
Kristie Bloom et al.
MOLECULAR THERAPY (2013)
Optical control of mammalian endogenous transcription and epigenetic states
Silvana Konermann et al.
NATURE (2013)
DNA targeting specificity of RNA-guided Cas9 nucleases
Patrick D. Hsu et al.
NATURE BIOTECHNOLOGY (2013)
Heritable gene targeting in the mouse and rat using a CRISPR-Cas system
Dali Li et al.
NATURE BIOTECHNOLOGY (2013)
The ATM protein kinase: regulating the cellular response to genotoxic stress, and more
Yosef Shiloh et al.
NATURE REVIEWS MOLECULAR CELL BIOLOGY (2013)
TMC1 and TMC2 Are Components of the Mechanotransduction Channel in Hair Cells of the Mammalian Inner Ear
Bifeng Pan et al.
NEURON (2013)
Transcription activator like effector (TALE)-directed piggyBac transposition in human cells
Jesse B. Owens et al.
NUCLEIC ACIDS RESEARCH (2013)
RNA-Guided Human Genome Engineering via Cas9
Prashant Mali et al.
SCIENCE (2013)
Multiplex Genome Engineering Using CRISPR/Cas Systems
Le Cong et al.
SCIENCE (2013)
Repair of Strand Breaks by Homologous Recombination
Maria Jasin et al.
COLD SPRING HARBOR PERSPECTIVES IN BIOLOGY (2013)
Production of Apolipoprotein C-III Knockout Rabbits using Zinc Finger Nucleases
Dongshan Yang et al.
JOVE-JOURNAL OF VISUALIZED EXPERIMENTS (2013)
A foundation for universal T-cell based immunotherapy: T cells engineered to express a CD19-specific chimeric-antigen-receptor and eliminate expression of endogenous TCR
Hiroki Torikai et al.
BLOOD (2012)
Life in the Fast Lane: Mammalian Disease Models in the Genomics Era
Lukas E. Dow et al.
CELL (2012)
Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer
Elena Provasi et al.
NATURE MEDICINE (2012)
Animal Models of Neurological Disorders
Marie-Francoise Chesselet et al.
NEUROTHERAPEUTICS (2012)
Chimeric piggyBac transposases for genomic targeting in human cells
Jesse B. Owens et al.
NUCLEIC ACIDS RESEARCH (2012)
Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria
Giedrius Gasiunas et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2012)
A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity
Martin Jinek et al.
SCIENCE (2012)
AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness
Jean Bennett et al.
SCIENCE TRANSLATIONAL MEDICINE (2012)
A TALE nuclease architecture for efficient genome editing
Jeffrey C. Miller et al.
NATURE BIOTECHNOLOGY (2011)
Revealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection
Vikram Pattanayak et al.
NATURE METHODS (2011)
Ex vivo gene transfer and correction for cell-based therapies
Luigi Naldini
NATURE REVIEWS GENETICS (2011)
Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting
Tomas Cermak et al.
NUCLEIC ACIDS RESEARCH (2011)
A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity
Claudio Mussolino et al.
NUCLEIC ACIDS RESEARCH (2011)
The Discovery of Zinc Fingers and Their Applications in Gene Regulation and Genome Manipulation
Aaron Klug
ANNUAL REVIEW OF BIOCHEMISTRY, VOL 79 (2010)
Xanthomonas AvrBs3 Family-Type III Effectors: Discovery and Function
Jens Boch et al.
ANNUAL REVIEW OF PHYTOPATHOLOGY, VOL 48 (2010)
Targeting DNA Double-Strand Breaks with TAL Effector Nucleases
Michelle Christian et al.
GENETICS (2010)
Zinc-finger Nucleases as a Novel Therapeutic Strategy for Targeting Hepatitis B Virus DNAs
Thomas J. Cradick et al.
MOLECULAR THERAPY (2010)
Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms
Akin Akinc et al.
MOLECULAR THERAPY (2010)
Precise targeted integration by a chimaeric transposase zinc-finger fusion protein
Xiaofeng Feng et al.
NUCLEIC ACIDS RESEARCH (2010)
Human RPE65 Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year
Artur V. Cideciyan et al.
HUMAN GENE THERAPY (2009)
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
Albert M. Maguire et al.
LANCET (2009)
Transposon-mediated genome manipulation in vertebrates
Zoltan Ivics et al.
NATURE METHODS (2009)
Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency.
Alessandro Aiuti et al.
NEW ENGLAND JOURNAL OF MEDICINE (2009)
Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors
Jens Boch et al.
SCIENCE (2009)
A Simple Cipher Governs DNA Recognition by TAL Effectors
Matthew J. Moscou et al.
SCIENCE (2009)
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
Salima Hacein-Bey-Abina et al.
JOURNAL OF CLINICAL INVESTIGATION (2008)
Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases
Elena E. Perez et al.
NATURE BIOTECHNOLOGY (2008)
ATR: an essential regulator of genome integrity
Karlene A. Cimprich et al.
NATURE REVIEWS MOLECULAR CELL BIOLOGY (2008)
Safety and efficacy of gene transfer for Leber's congenital amaurosis
Albert M. Maguire et al.
NEW ENGLAND JOURNAL OF MEDICINE (2008)
Effect of gene therapy on visual function in Leber's congenital amaurosis
James W. B. Bainbridge et al.
NEW ENGLAND JOURNAL OF MEDICINE (2008)
CRISPR provides acquired resistance against viruses in prokaryotes
Rodolphe Barrangou et al.
SCIENCE (2007)
Mutations in the CEP290 (NPHP6) gene are a frequent cause of leber congenital amaurosis
Anneke I. den Hollander et al.
AMERICAN JOURNAL OF HUMAN GENETICS (2006)
Clustered regularly interspaced short palindrome repeats (CRISPRs) have spacers of extrachromosomal origin
A Bolotin et al.
MICROBIOLOGY-SGM (2005)
Highly efficient endogenous human gene correction using designed zinc-finger nucleases
FD Urnov et al.
NATURE (2005)
CRISPR elements in Yersinia pestis acquire new repeats by preferential uptake of bacteriophage DNA, and provide additional tools for evolutionary studies
C Pourcel et al.
MICROBIOLOGY-SGM (2005)
Intervening sequences of regularly spaced prokaryotic repeats derive from foreign genetic elements
FJM Mojica et al.
JOURNAL OF MOLECULAR EVOLUTION (2005)
Retroviral restriction by APOBEC proteins
RS Harris et al.
NATURE REVIEWS IMMUNOLOGY (2004)
Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer
SE Raper et al.
MOLECULAR GENETICS AND METABOLISM (2003)
Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning
A Aiuti et al.
SCIENCE (2002)
Engineering polydactyl zinc-finger transcription factors
RR Beerli et al.
NATURE BIOTECHNOLOGY (2002)
Share Paper
