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Arthur W. Nienhuis et al.
MOLECULAR THERAPY (2017)
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Hui-Ying Xue et al.
JOURNAL OF MEDICAL GENETICS (2016)
Targeting of the human F8 at the multicopy rDNA locus in Hemophilia a patient-derived iPSCs using TALENickases
Jialun Pang et al.
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Yuting Guan et al.
EMBO MOLECULAR MEDICINE (2016)
Identification of mutations in the F8 and F9 gene in families with haemophilia using targeted high-throughput sequencing
C. Lyu et al.
HAEMOPHILIA (2016)
Engineering the AAVS1 locus for consistent and scalable transgene expression in human iPSCs and their differentiated derivatives
Fabian Oceguera-Yanez et al.
METHODS (2016)
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Chul-Yong Park et al.
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Alan Trounson et al.
NATURE REVIEWS MOLECULAR CELL BIOLOGY (2016)
Generation of a TLE3 heterozygous knockout human embryonic stem cell line using CRISPR-Cas9
Anne M. Bara et al.
STEM CELL RESEARCH (2016)
Genome engineering in the yeast pathogen Candida glabrata using the CRISPR-Cas9 system
Ludovic Enkler et al.
SCIENTIFIC REPORTS (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)
Different Effects of sgRNA Length on CRISPR-mediated Gene Knockout Efficiency
Jian-Ping Zhang et al.
SCIENTIFIC REPORTS (2016)
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Chul-Yong Park et al.
CELL STEM CELL (2015)
Production of Gene-Corrected Adult Beta Globin Protein in Human Erythrocytes Differentiated From Patient iPSCs After Genome Editing of the Sickle Point Mutation
Xiaosong Huang et al.
STEM CELLS (2015)
Improved Hematopoietic Differentiation Efficiency of Gene-Corrected Beta-Thalassemia Induced Pluripotent Stem Cells by CRISPR/Cas9 System
Bing Song et al.
STEM CELLS AND DEVELOPMENT (2015)
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)
Whole-Genome Sequencing Analysis Reveals High Specificity of CRISPR/Cas9 and TALEN-Based Genome Editing in Human iPSCs
Cory Smith et al.
CELL STEM CELL (2014)
Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac
Fei Xie et al.
GENOME RESEARCH (2014)
Modeling of hemophilia A using patient-specific induced pluripotent stem cells derived from urine cells
Bei Jia et al.
LIFE SCIENCES (2014)
Sequence-specific antimicrobials using efficiently delivered RNA-guided nucleases
Robert J. Citorik et al.
NATURE BIOTECHNOLOGY (2014)
Genome-wide analysis reveals characteristics of off-target sites bound by the Cas9 endonuclease
Cem Kuscu et al.
NATURE BIOTECHNOLOGY (2014)
Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
A. C. Nathwani et al.
NEW ENGLAND JOURNAL OF MEDICINE (2014)
TALENs facilitate targeted genome editing in human cells with high specificity and low cytotoxicity
Claudio Mussolino et al.
NUCLEIC ACIDS RESEARCH (2014)
Genetic Screens in Human Cells Using the CRISPR-Cas9 System
Tim Wang et al.
SCIENCE (2014)
A Simple and Efficient System for Regulating Gene Expression in Human Pluripotent Stem Cells and Derivatives
Kun Qian et al.
STEM CELLS (2014)
Stable Enhanced Green Fluorescent Protein Expression After Differentiation and Transplantation of Reporter Human Induced Pluripotent Stem Cells Generated by AAVS1 Transcription Activator-Like Effector Nucleases
Yongquan Luo et al.
STEM CELLS TRANSLATIONAL MEDICINE (2014)
Reprogrammed Cells for Disease Modeling and Regenerative Medicine
Anne B. C. Cherry et al.
ANNUAL REVIEW OF MEDICINE, VOL 64 (2013)
Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients
Gerald Schwank et al.
CELL STEM CELL (2013)
DNA targeting specificity of RNA-guided Cas9 nucleases
Patrick D. Hsu et al.
NATURE BIOTECHNOLOGY (2013)
High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity
Vikram Pattanayak et al.
NATURE BIOTECHNOLOGY (2013)
High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells
Yanfang Fu et al.
NATURE BIOTECHNOLOGY (2013)
Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells
Maarten Holkers 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)
An Efficient Nonviral Method to Generate Integration-Free Human-Induced Pluripotent Stem Cells from Cord Blood and Peripheral Blood Cells
Keisuke Okita et al.
STEM CELLS (2013)
Cellular Reprogramming of Human Peripheral Blood Cells
Xiao-Bing Zhang
GENOMICS PROTEOMICS & BIOINFORMATICS (2013)
Derivation of autism spectrum disorder-specific induced pluripotent stem cells from peripheral blood mononuclear cells
Brooke A. DeRosa et al.
NEUROSCIENCE LETTERS (2012)
Efficient human iPS cell derivation by a non-integrating plasmid from blood cells with unique epigenetic and gene expression signatures
Bin-Kuan Chou et al.
CELL RESEARCH (2011)
Site-specific integration and tailoring of cassette design for sustainable gene transfer
Angelo Lombardo et al.
NATURE METHODS (2011)
Reprogramming of Human Peripheral Blood Cells to Induced Pluripotent Stem Cells
Judith Staerk et al.
CELL STEM CELL (2010)
Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome
Russell C. DeKelver et al.
GENOME RESEARCH (2010)
Highly Efficient Generation of Human Hepatocyte-Like Cells from Induced Pluripotent Stem Cells
Karim Si-Tayeb et al.
HEPATOLOGY (2010)
Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases
Dirk Hockemeyer et al.
NATURE BIOTECHNOLOGY (2009)
Induced pluripotent stem cell lines derived from human somatic cells
Junying Yu et al.
SCIENCE (2007)
Induction of pluripotent stem cells from adult human fibroblasts by defined factors
Kazutoshi Takahashi et al.
CELL (2007)
Identification of an insulator in AAVS1, a preferred region for integration of adeno-associated virus DNA
T Ogata et al.
JOURNAL OF VIROLOGY (2003)