4.6 Review

Advances in CRISPR therapeutics

Related references

Note: Only part of the references are listed.
Article Biochemistry & Molecular Biology

Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins

Samagya Banskota et al.

Summary: We have developed and applied engineered DNA-free virus-like particles (eVLPs) for efficient delivery of gene editing tools, achieving high levels of base editing. By overcoming the limitations of cargo packaging, release, and localization, the fourth-generation eVLPs demonstrated efficient base editing in mouse and human cells. In addition, the off-target effects of eVLPs were significantly reduced compared to other delivery methods such as AAV or plasmid.
Article Biotechnology & Applied Microbiology

Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing

Andrew Anzalone et al.

Summary: The twin prime editing (twinPE) method described in this study allows for programmable replacement or excision of DNA sequences at endogenous human genomic sites without the need for double-strand DNA breaks (DSBs). When combined with a site-specific serine recombinase, twinPE enables targeted integration of gene-sized DNA plasmids (>5,000 bp) and targeted sequence inversions of 40 kb in human cells. TwinPE expands the capabilities of precision gene editing and may synergize with other tools for the correction or complementation of large or complex human pathogenic alleles.

NATURE BIOTECHNOLOGY (2022)

Article Biochemistry & Molecular Biology

CRISPR interference and activation of the microRNA-3662-HBP1 axis control progression of triple-negative breast cancer

Baozhu Yi et al.

Summary: MiR-3662 is minimally expressed in normal tissues but highly expressed in various cancers, especially in TNBC and African American breast cancer. Inhibition or knockout of miR-3662 suppresses proliferation and migration of TNBC cells by reducing Wnt/β-catenin signaling activation. HMG-box transcription factor 1 (HBP-1) is identified as a target of miR-3662 responsible for TNBC cell proliferation.

ONCOGENE (2022)

Article Biotechnology & Applied Microbiology

Dual-AAV delivering split prime editor system for in vivo genome editing

Shengyao Zhi et al.

Summary: Prime editor (PE) is a new genome editing tool that has the potential to correct the majority of known human genetic disease-related mutations. In this study, split-PEs were constructed and delivered using dual adenoassociated viruses (AAVs), successfully mediating gene editing in human cells and adult mouse retina.

MOLECULAR THERAPY (2022)

Article Cell Biology

Multiplexed genome regulation in vivo with hyper-efficient Cas12a

Lucie Y. Guo et al.

Summary: Through protein engineering, a highly efficient Lachnospiraceae bacterium Cas12a variant, called hyperCas12a, has been developed to enhance gene editing and activation efficiency, and applied for multiplex gene activation in the mouse retina. The hyperCas12a system offers a versatile tool for gene modulation and gene therapy applications.

NATURE CELL BIOLOGY (2022)

Article Multidisciplinary Sciences

Broad-spectrum CRISPR-mediated inhibition of SARS-CoV-2 variants and endemic coronaviruses in vitro

Leiping Zeng et al.

Summary: The authors demonstrate that CRISPR-Cas13d can effectively inhibit a wide range of human coronaviruses, including new SARS-CoV-2 variants, in combination with small molecule drugs. The CRISPR-Cas13d system reduces viral titers by over 99% and enhances the therapeutic effects of different antiviral drugs. Lipid nanoparticle-mediated RNA delivery enables the Cas13d system to treat infections from multiple coronavirus variants in human primary airway epithelium cultures. This study establishes CRISPR-Cas13 as a broad-spectrum antiviral strategy that complements existing vaccination and antiviral treatment strategies.

NATURE COMMUNICATIONS (2022)

Review Biochemistry & Molecular Biology

Therapeutic in vivo delivery of gene editing agents

Aditya Raguram et al.

Summary: In vivo gene editing therapies have the potential to treat the underlying causes of genetic diseases. This review examines current delivery technologies for therapeutic in vivo gene editing, including viral vectors, lipid nanoparticles, and virus-like particles. The benefits and drawbacks of each method are compared, and opportunities for future improvements are highlighted.
Article Biotechnology & Applied Microbiology

Optimization of AsCas12a for combinatorial genetic screens in human cells

Peter C. DeWeirdt et al.

Summary: In this study, an optimized Cas12a toolkit was developed for pooled, combinatorial genetic screens in human cells. By assaying the activity of thousands of guides, refined on-target design rules and comprehensive off-target rules were established. The toolkit was validated by screening for synthetic lethality in cancer cells and showed similar performance to Cas9 in genome-wide dropout screens with greatly reduced library size.

NATURE BIOTECHNOLOGY (2021)

Review Biotechnology & Applied Microbiology

Directed Evolution of CRISPR/Cas Systems for Precise Gene Editing

Rongming Liu et al.

Summary: CRISPR technology, a universal tool for genome engineering, has been revolutionizing biotechnology. The diverse Cas proteins with complex structures contain multiple evolutionarily conserved functional domains, and engineering key domains can modify the functions of CRISPR/Cas systems.

TRENDS IN BIOTECHNOLOGY (2021)

Article Multidisciplinary Sciences

In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice

Luke W. Koblan et al.

Summary: Using adenine base editor (ABE), researchers successfully corrected the pathogenic mutation in fibroblasts from children with Hutchinson-Gilford progeria syndrome, mitigating RNA mis-splicing, reducing progerin levels, and correcting nuclear abnormalities. In vivo base editing with AAV9 encoding ABE in transgenic mice resulted in substantial, durable correction of the mutation, restoration of normal RNA splicing, and improvement of vascular pathology, ultimately extending the lifespan of the mice. These findings demonstrate the potential of in vivo base editing as a treatment for genetic diseases.

NATURE (2021)

Article Biotechnology & Applied Microbiology

Targeting herpes simplex virus with CRISPR-Cas9 cures herpetic stromal keratitis in mice

Di Yin et al.

Summary: The study targeted HSV-1 genomes directly using mRNA-carrying lentiviral particles (HELP), which efficiently blocked HSV-1 replication and reduced the occurrence of HSK in three different infection models. HELP was capable of eliminating the viral reservoir via retrograde transport from corneas to trigeminal ganglia, while inhibiting viral replication in human-derived corneas without causing off-target effects.

NATURE BIOTECHNOLOGY (2021)

Review Cell Biology

CRISPR technologies for precise epigenome editing

Muneaki Nakamura et al.

Summary: The review discusses CRISPR-based epigenome engineering technologies for modulating histone and DNA modifications, perturbing DNA and RNA regulatory elements, and chromatin organization. The epigenome involves complex cellular processes governing genomic activity. Repurposing prokaryotic CRISPR systems has enabled the development of diverse technologies in epigenome engineering.

NATURE CELL BIOLOGY (2021)

Article Medicine, General & Internal

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

H. Frangoul et al.

Summary: Research utilizing CRISPR-Cas9 technology for gene editing in patients with TDT and SCD showed high levels of edited alleles in bone marrow and blood, leading to partial or complete elimination of symptoms.

NEW ENGLAND JOURNAL OF MEDICINE (2021)

Article Engineering, Biomedical

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.

Summary: The study demonstrates the efficacy of engineered lentiviruses co-packaging SpCas9 mRNA and a guide RNA targeting the Vegfa gene in preventing wet age-related macular degeneration in mice, providing a potential new treatment approach for retinal neovascular diseases.

NATURE BIOMEDICAL ENGINEERING (2021)

Article Medicine, Research & Experimental

CRISPR/Cas9 directed to the Ube3a antisense transcript improves Angelman syndrome phenotype in mice

Ralf S. Schmid et al.

Summary: Gene editing technology shows potential in treating Angelman syndrome in a mouse model by partially correcting behavioral phenotypes. This therapeutic approach aims to unsilence the paternal gene in neurons, providing lasting therapeutic effects for patients with AS.

JOURNAL OF CLINICAL INVESTIGATION (2021)

Article Pharmacology & Pharmacy

A CRISPR-Cas9 repressor for epigenetic silencing of KRAS

Jingwen Liu et al.

Summary: The research team successfully designed a new strategy by combining CRISPR-Cas9 technology with HDAC1 epigenetic silencing to silence KRAS and inhibit the growth and proliferation of cancer cells. This strategy holds promise as a potential effective approach for treating cancers driven by KRAS mutations in the future.

PHARMACOLOGICAL RESEARCH (2021)

Review Ophthalmology

LEBER CONGENITAL AMAUROSIS DUE TO CEP290 MUTATIONS-SEVERE VISION IMPAIRMENT WITH A HIGH UNMET MEDICAL NEED A Review

Bart P. Leroy et al.

Summary: LCA10, caused by CEP290 mutations, is a genetic retinal disease leading to severe visual impairment in early childhood. There are currently no approved treatments, but investigational strategies include RNA editing using antisense oligonucleotides or CRISPR-based approaches. The antisense oligonucleotide therapy QR-110 has shown promising results in early trials, with ongoing phase 3 clinical trials.

RETINA-THE JOURNAL OF RETINAL AND VITREOUS DISEASES (2021)

Article Cell Biology

Long-lasting analgesia via targeted in situ repression of NaV1.7 in mice

Ana M. Moreno et al.

Summary: This study demonstrated successful targeted repression of Nav1.7 in mouse pain models through epigenome engineering approaches, leading to significant reduction in thermal hyperalgesia in inflammatory states and tactile allodynia in neuropathic pain without affecting normal motor function. The long-lasting analgesia achieved through this in vivo epigenetic repression of Nav1.7 methodology, named pain LATER, holds therapeutic potential in management of persistent pain states.

SCIENCE TRANSLATIONAL MEDICINE (2021)

Article Medicine, Research & Experimental

Targeted epigenetic repression by CRISPR/dSaCas9 suppresses pathogenic DUX4-fl expression in FSHD

Charis L. Himeda et al.

Summary: Researchers have re-engineered a platform to deliver more potent epigenetic repressors for the treatment of FSHD, effectively targeting and repressing DUX4 in skeletal muscles. This new approach has potential for clinical use of dCas9-based chromatin effectors in skeletal muscle disorders.

MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2021)

Article Biochemistry & Molecular Biology

CRISPR-CasRx Targeting LncRNA LINC00341 Inhibits Tumor Cell Growth in vitro and in vivo

Chunjing Li et al.

Summary: The study revealed that LINC00341 is overexpressed in bladder cancer tissues compared to healthy tissues, with higher levels in high-grade tumors. Inhibition of LINC00341 resulted in reduced cell proliferation and migration, and increased apoptosis. Additionally, silencing LINC00341 upregulated the expressions of p21, Bax, and E-cadherin.

FRONTIERS IN MOLECULAR BIOSCIENCES (2021)

Article Biochemistry & Molecular Biology

Genome-wide programmable transcriptional memory by CRISPR-based epigenome editing

James K. Nunez et al.

Summary: CRISPRoff is a programmable epigenetic memory writer that can heritably alter gene expression by initiating specific DNA methylation and gene repression. The tool utilizes a single dead Cas9 fusion protein to establish these modifications, which are maintained through cell division and differentiation processes. By pairing CRISPRoff with genome-wide screens and analysis of chromatin marks, rules for heritable gene silencing can be established, with the tool capable of targeting a wide range of genes beyond canonical CpG islands.
Article Multidisciplinary Sciences

Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice

Pengpeng Liu et al.

Summary: The study introduces an NLS-optimized SpCas9-based prime editor that enhances genome editing efficiency and demonstrates the potential to induce tumor formation and correct pathogenic mutations in adult mice through somatic cell editing. The use of dual adeno-associated virus (AAVs) for delivery of a split-intein prime editor further establishes the capability of this system for in vivo installation of sequence modifications with important implications for disease modeling and correction.

NATURE COMMUNICATIONS (2021)

Article Multidisciplinary Sciences

Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing

F. Chemello et al.

Summary: This study demonstrated the use of adenine base editor to restore dystrophin expression in cardiomyocytes affected by Duchenne muscular dystrophy mutation, as well as reframing the dystrophin open reading frame. In vivo experiments with mice showed successful gene editing and disease correction, indicating the potential of nucleotide editing for diverse DMD mutations with minimal genome modification.

SCIENCE ADVANCES (2021)

Article Engineering, Biomedical

CRISPR activation of long non-coding RNA DANCR promotes bone regeneration

Nuong Thi Kieu Nguyen et al.

Summary: Healing of large calvarial bone defects in adults is challenging and typically follows the intramembranous pathway. Implanting adipose-derived stem cells (ASC) that differentiate towards chondrogenic lineage can enhance bone repair. Activation of the long non-coding RNA DANCR by SadCas9-VPR-based CRISPRa improves rASC chondrogenesis and promotes calvarial bone healing in rats.

BIOMATERIALS (2021)

Article Biochemistry & Molecular Biology

Engineered miniature CRISPR-Cas system for mammalian genome regulation and editing

Xiaoshu Xu et al.

Summary: Researchers have developed a miniature Cas system, CasMINI, that can achieve high levels of gene activation in mammalian cells and has comparable activity and high specificity to Cas12a, making it suitable for gene editing and gene therapy applications.

MOLECULAR CELL (2021)

Article Multidisciplinary Sciences

Base editing of haematopoietic stem cells rescues sickle cell disease in mice

Gregory A. Newby et al.

Summary: The study demonstrated successful conversion of sickle cell disease allele into a non-pathogenic variant using adenine base editor, with durable therapeutic effects. The edited HSPCs improved physiological parameters and reduced pathological abnormalities in spleens of mice, indicating the potential for long-lasting and effective treatment for SCD.

NATURE (2021)

Article Biochemical Research Methods

Transgenic mice for in vivo epigenome editing with CRISPR-based systems

Matthew Gemberling et al.

Summary: Two conditional transgenic mouse lines based on CRISPRa and CRISPRi enable epigenome editing in vivo.

NATURE METHODS (2021)

Article Medicine, General & Internal

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

Julian D. Gillmore et al.

Summary: The gene-editing therapeutic agent NTLA-2001 effectively reduced serum TTR protein concentrations in patients with ATTR amyloidosis, showing potential safety and pharmacodynamic effects in clinical studies.

NEW ENGLAND JOURNAL OF MEDICINE (2021)

Article Cell Biology

Targeted delivery of CRISPR-Cas9 and transgenes enables complex immune cell engineering

Jennifer R. Hamilton et al.

Summary: A new strategy has been developed in this study, which uses engineered lentiviral particles to introduce Cas9 RNPs and a CAR transgene into human T cells ex vivo, allowing targeting of specific cell types within a mixed population. This approach provides a method for genetic modification of immune cells without the need for electroporation.

CELL REPORTS (2021)

Review Cell Biology

Programmable System of Cas13-Mediated RNA Modification and Its Biological and Biomedical Applications

Tian Tang et al.

Summary: CRISPR-Cas13 is a versatile tool for controlling gene expression and cell fate at the RNA level, in addition to RNA interference, it can also modulate various aspects of RNA fate through RNA modifications. These toolkits have been applied in cell biology, disease, and imaging, but potential limitations such as off-target editing, low efficiency, and delivery restrictions exist.

FRONTIERS IN CELL AND DEVELOPMENTAL BIOLOGY (2021)

Review Medicine, Research & Experimental

Progress and challenges in CRISPR-mediated therapeutic genome editing for monogenic diseases

Colin T. Konishi et al.

Summary: CRISPR/Cas genome editing tools offer personalized treatment approaches for monogenic diseases, with broad and multifaceted therapeutic applications targeting diverse diseases.

JOURNAL OF BIOMEDICAL RESEARCH (2021)

Review Biotechnology & Applied Microbiology

Base and Prime Editing Technologies for Blood Disorders

Panagiotis Antoniou et al.

Summary: Base editing, as a promising genome editing technology, allows the introduction of point mutations without generating double strand breaks, making it a potential therapeutic strategy for genetic diseases such as blood disorders. With two major classes of base editors developed, allowing C>T and A>G conversions, this technology offers advantages such as high efficiency, specificity, and low rate of InDels.

FRONTIERS IN GENOME EDITING (2021)

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Multiple Input Sensing and Signal Integration Using a Split Cas12a System

Hannah R. Kempton et al.

MOLECULAR CELL (2020)

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Treatment of a Mouse Model of ALS by In Vivo Base Editing

Colin K. W. Lim et al.

MOLECULAR THERAPY (2020)

Review Microbiology

Evolutionary classification of CRISPR-Cas systems: a burst of class 2 and derived variants

Kira S. Makarova et al.

NATURE REVIEWS MICROBIOLOGY (2020)

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Noncoding mutations target cis-regulatory elements of the FOXA1 plexus in prostate cancer

Stanley Zhou et al.

NATURE COMMUNICATIONS (2020)

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Endogenous Type I CRISPR-Cas: From Foreign DNA Defense to Prokaryotic Engineering

Zheng Yanli et al.

FRONTIERS IN BIOENGINEERING AND BIOTECHNOLOGY (2020)

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Gene editing and CRISPR in the clinic: current and future perspectives

Matthew P. Hirakawa et al.

BIOSCIENCE REPORTS (2020)

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Development of CRISPR as an Antiviral Strategy to Combat SARS-CoV-2 and Influenza

Timothy R. Abbott et al.

Article Biotechnology & Applied Microbiology

CRISPR/Cas9-Mediated miR-29b Editing as a Treatment of Different Types of Muscle Atrophy in Mice

Jin Li et al.

MOLECULAR THERAPY (2020)

Article Biotechnology & Applied Microbiology

Directed evolution of adenine base editors with increased activity and therapeutic application

Nicole M. Gaudelli et al.

NATURE BIOTECHNOLOGY (2020)

Article Biotechnology & Applied Microbiology

Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity

Michelle F. Richter et al.

NATURE BIOTECHNOLOGY (2020)

Article Biotechnology & Applied Microbiology

Dual base editor catalyzes both cytosine and adenine base conversions in human cells

Xiaohui Zhang et al.

NATURE BIOTECHNOLOGY (2020)

Article Biotechnology & Applied Microbiology

A dual-deaminase CRISPR base editor enables concurrent adenine and cytosine editing

Julian Grunewald et al.

NATURE BIOTECHNOLOGY (2020)

Review Biochemistry & Molecular Biology

Recent Advances in CRISPR/Cas9 Delivery Strategies

Bon Ham Yip

BIOMOLECULES (2020)

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Extinction of all infectious HIV in cell culture by the CRISPR-Cas12a system with only a single crRNA

Zongliang Gao et al.

NUCLEIC ACIDS RESEARCH (2020)

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Intronic CRISPR Repair in a Preclinical Model of Noonan Syndrome-Associated Cardiomyopathy

Ulrich Hanses et al.

CIRCULATION (2020)

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Determinants of Base Editing Outcomes from Target Library Analysis and Machine Learning

Mandana Arbab et al.

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Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors

Andrew V. Anzalone et al.

NATURE BIOTECHNOLOGY (2020)

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Cas9 gene therapy for Angelman syndrome trapsUbe3a-ATSlong non-coding RNA

Justin M. Wolter et al.

NATURE (2020)

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Base editing: advances and therapeutic opportunities

Elizabeth M. Porto et al.

NATURE REVIEWS DRUG DISCOVERY (2020)

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In vivo CRISPR/Cas9 targeting of fusion oncogenes for selective elimination of cancer cells

M. Martinez-Lage et al.

NATURE COMMUNICATIONS (2020)

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CRISPR based editing of SIV proviral DNA in ART treated non-human primates

Pietro Mancuso et al.

NATURE COMMUNICATIONS (2020)

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Genome Editing-Mediated Utrophin Upregulation in Duchenne Muscular Dystrophy Stem Cells

Kasturi Sengupta et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2020)

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CRISPR-CasΦ from huge phages is a hypercompact genome editor

Patrick Pausch et al.

SCIENCE (2020)

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Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases

Shao-Shuai Wu et al.

THERANOSTICS (2020)

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CasX enzymes comprise a distinct family of RNA-guided genome editors

Jun-Jie Liu et al.

NATURE (2019)

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Activating PTEN Tumor Suppressor Expression with the CRISPR/dCas9 System

Colette Moses et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2019)

Article Biochemistry & Molecular Biology

CRISPR-mediated upregulation of DR5 and downregulation of cFLIP synergistically sensitize HeLa cells to TRAIL-mediated apoptosis

Naresh Poondla et al.

BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS (2019)

Article Biochemistry & Molecular Biology

Introducing a Spectrum of Long-Range Genomic Deletions in Human Embryonic Stem Cells Using Type I CRISPR-Cas

Adam E. Dolan et al.

MOLECULAR CELL (2019)

Article Multidisciplinary Sciences

A mutation-independent approach for muscular dystrophy via upregulation of a modifier gene

Dwi U. Kemaladewi et al.

NATURE (2019)

Article Multidisciplinary Sciences

Transposon-encoded CRISPR-Cas systems direct RNA-guided DNA integration

Sanne E. Klompe et al.

NATURE (2019)

Review Clinical Neurology

Hereditary transthyretin amyloidosis: a model of medical progress for a fatal disease

David Adams et al.

NATURE REVIEWS NEUROLOGY (2019)

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RNA-guided DNA insertion with CRISPR-associated transposases

Jonathan Strecker et al.

SCIENCE (2019)

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Multiplexed genome engineering by Cas12a and CRISPR arrays encoded on single transcripts

Carlo C. Campa et al.

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The Role of Non-coding RNAs in Oncology

Frank J. Slack et al.

Article Multidisciplinary Sciences

Search-and-replace genome editing without double-strand breaks or donor DNA

Andrew V. Anzalone et al.

NATURE (2019)

Review Biotechnology & Applied Microbiology

Era of Genomic Medicine: A Narrative Review on CRISPR Technology as a Potential Therapeutic Tool for Human Diseases

Odatha W. Kotagama et al.

BIOMED RESEARCH INTERNATIONAL (2019)

Article Biochemistry & Molecular Biology

Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10

Morgan L. Maeder et al.

NATURE MEDICINE (2019)

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Functionally diverse type V CRISPR-Cas systems

Winston X. Yan et al.

SCIENCE (2019)

Article Multidisciplinary Sciences

Engineering of CRISPR-Cas12b for human genome editing

Jonathan Strecker et al.

NATURE COMMUNICATIONS (2019)

Review Biochemistry & Molecular Biology

Development of Novel Therapeutic Agents by Inhibition of Oncogenic MicroRNAs

Dinh-Duc Nguyen et al.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2018)

Article Biochemistry & Molecular Biology

LncRNA THOR promotes human renal cell carcinoma cell growth

Xue-ting Ye et al.

BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS (2018)

Article Biochemistry & Molecular Biology

Transcriptome Engineering with RNA-Targeting Type VI-D CRISPR Effectors

Silvana Konermann et al.

Article Biochemistry & Molecular Biology

Rescue of Fragile X Syndrome Neurons by DNA Methylation Editing of the FMR1 Gene

X. Shawn Liu et al.

Article Pharmacology & Pharmacy

Delivering CRISPR: a review of the challenges and approaches

Christopher A. Lino et al.

DRUG DELIVERY (2018)

Article Biochemistry & Molecular Biology

CRISPR/Cas9-mediated knockout of HBsAg inhibits proliferation and tumorigenicity of HBV-positive hepatocellular carcinoma cells

Jia Song et al.

JOURNAL OF CELLULAR BIOCHEMISTRY (2018)

Article Biochemistry & Molecular Biology

Activation of the Anti-Aging and Cognition-Enhancing Gene Klotho by CRISPR-dCas9 Transcriptional Effector Complex

Ci-Di Chen et al.

JOURNAL OF MOLECULAR NEUROSCIENCE (2018)

Article Biotechnology & Applied Microbiology

In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation

Ana M. Moreno et al.

MOLECULAR THERAPY (2018)

Article Biotechnology & Applied Microbiology

Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy

Seuk-Min Ryu et al.

NATURE BIOTECHNOLOGY (2018)

Article Biotechnology & Applied Microbiology

Improving cytidine and adenine base editors by expression optimization and ancestral reconstruction

Luke W. Koblan et al.

NATURE BIOTECHNOLOGY (2018)

Article Medicine, Research & Experimental

Epigenetic Targeting of Granulin in Hepatoma Cells by Synthetic CRISPR dCas9 Epi-suppressors

Hong Wang et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2018)

Review Biotechnology & Applied Microbiology

Strategies for controlling CRISPR/Cas9 off-target effects and biological variations in mammalian genome editing experiments

Michelle L. Kimberland et al.

JOURNAL OF BIOTECHNOLOGY (2018)

Article Biotechnology & Applied Microbiology

Therapeutic Genome Editing for Myotonic Dystrophy Type 1 Using CRISPR/Cas9

Yanlin Wang et al.

MOLECULAR THERAPY (2018)

Article Multidisciplinary Sciences

Evolved Cas9 variants with broad PAM compatibility and high DNA specificity

Johnny H. Hu et al.

NATURE (2018)

Article Biochemistry & Molecular Biology

Treatment of a metabolic liver disease by in vivo genome base editing in adult mice

Lukas Villiger et al.

NATURE MEDICINE (2018)

Article Multidisciplinary Sciences

Programmed DNA destruction by miniature CRISPR-Cas14 enzymes

Lucas B. Harrington et al.

SCIENCE (2018)

Article Cardiac & Cardiovascular Systems

A Precision Medicine Approach to the Rescue of Function on Malignant Calmodulinopathic Long-QT Syndrome

Worawan B. Limpitikul et al.

CIRCULATION RESEARCH (2017)

Article Multidisciplinary Sciences

New CRISPR-Cas systems from uncultivated microbes

David Burstein et al.

NATURE (2017)

Article Biochemistry & Molecular Biology

Efficient targeted DNA methylation with chimeric dCas9-Dnmt3a-Dnmt3L methyltransferase

Peter Stepper et al.

NUCLEIC ACIDS RESEARCH (2017)

Article Biochemistry & Molecular Biology

CRISPR-Cas9 Mediated Gene-Silencing of the Mutant Huntingtin Gene in an In Vitro Model of Huntington's Disease

Nivya Kolli et al.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2017)

Article Biochemistry & Molecular Biology

Linc-RoR promotes MAPK/ERK signaling and confers estrogen-independent growth of breast cancer

Wan-xin Peng et al.

MOLECULAR CANCER (2017)

Article Biotechnology & Applied Microbiology

Genome Editing Reveals Glioblastoma Addiction to MicroRNA-10b

Rachid El Fatimy et al.

MOLECULAR THERAPY (2017)

Article Biotechnology & Applied Microbiology

CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo

Alex Mas Monteys et al.

MOLECULAR THERAPY (2017)

Article Multidisciplinary Sciences

Programmable base editing of A.T to G.C in genomic DNA without DNA cleavage

Nicole M. Gaudelli et al.

NATURE (2017)

Article Biotechnology & Applied Microbiology

Increasing the genome-targeting scope and precision of base editing with engineered Cas9-cytidine deaminase fusions

Y. Bill Kim et al.

NATURE BIOTECHNOLOGY (2017)

Review Cell Biology

Non-homologous DNA end joining and alternative pathways to double-strand break repair

Howard H. Y. Chang et al.

NATURE REVIEWS MOLECULAR CELL BIOLOGY (2017)

Review Medicine, General & Internal

Sickle Cell Disease

Frederic B. Piel et al.

NEW ENGLAND JOURNAL OF MEDICINE (2017)

Article Cell Biology

Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy

Leonela Amoasii et al.

SCIENCE TRANSLATIONAL MEDICINE (2017)

Article Genetics & Heredity

Stabilization of Foxp3 expression by CRISPR-dCas9-based epigenome editing in mouse primary T cells

Masahiro Okada et al.

EPIGENETICS & CHROMATIN (2017)

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LncRNA AK023948 is a positive regulator of AKT

Pratirodh Koirala et al.

NATURE COMMUNICATIONS (2017)

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miR-29b contributes to multiple types of muscle atrophy

Jin Li et al.

NATURE COMMUNICATIONS (2017)

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Increased Expression of Laminin Subunit Alpha 1 Chain by dCas9-VP160

Arnaud Perrin et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2017)

Article Biochemistry & Molecular Biology

In Vivo Target Gene Activation via CRISPR/Cas9-Mediated Trans-epigenetic Modulation

Hsin-Kai Liao et al.

Article Genetics & Heredity

Spell Checking Nature: Versatility of CRISPR/Cas9 for Developing Treatments for Inherited Disorders

Dania Wojtal et al.

AMERICAN JOURNAL OF HUMAN GENETICS (2016)

Article Biochemistry & Molecular Biology

Inheritable Silencing of Endogenous Genes by Hit-and-Run Targeted Epigenetic Editing

Angelo Amabile et al.

Article Biochemistry & Molecular Biology

CRISPR-on system for the activation of the endogenous human INS gene

C. A. Gimenez et al.

GENE THERAPY (2016)

Article Biotechnology & Applied Microbiology

Complete Spectrum of CRISPR/Cas9-induced Mutations on HBV cccDNA

Christoph Seeger et al.

MOLECULAR THERAPY (2016)

Article Multidisciplinary Sciences

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

Alexis C. Komor et al.

NATURE (2016)

Article Biotechnology & Applied Microbiology

Targeted DNA demethylation in vivo using dCas9-peptide repeat and scFv-TET1 catalytic domain fusions

Sumiyo Morita et al.

NATURE BIOTECHNOLOGY (2016)

Review Clinical Neurology

Angelman syndrome - insights into a rare neurogenetic disorder

Karin Buiting et al.

NATURE REVIEWS NEUROLOGY (2016)

Article Biochemistry & Molecular Biology

Repurposing the CRISPR-Cas9 system for targeted DNA methylation

Aleksandar Vojta et al.

NUCLEIC ACIDS RESEARCH (2016)

Review Genetics & Heredity

Lateral Thinking: How Histone Modifications Regulate Gene Expression

Moyra Lawrence et al.

TRENDS IN GENETICS (2016)

Article Cell Biology

A CRISPR-based approach for targeted DNA demethylation

Xingxing Xu et al.

CELL DISCOVERY (2016)

Article Multidisciplinary Sciences

Elimination of HIV-1 Genomes from Human T-lymphoid Cells by CRISPR/Cas9 Gene Editing

Rafal Kaminski et al.

SCIENTIFIC REPORTS (2016)

Article Multidisciplinary Sciences

Precision Modulation of Neurodegenerative Disease-Related Gene Expression in Human iPSC-Derived Neurons

Sabrina Mahalia Heman-Ackah et al.

SCIENTIFIC REPORTS (2016)

Article Pharmacology & Pharmacy

Targeting hepatitis B virus cccDNA by CRISPR/Cas9 nuclease efficiently inhibits viral replication

Chunsheng Dong et al.

ANTIVIRAL RESEARCH (2015)

Article Biochemistry & Molecular Biology

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