Published in Stem Cell Reports on November 26, 2014
In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy. Science (2015) 4.18
Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy. Science (2015) 2.97
A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells. Cell Stem Cell (2016) 1.60
Genome-editing Technologies for Gene and Cell Therapy. Mol Ther (2016) 1.41
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CRISPR-mediated genotypic and phenotypic correction of a chronic granulomatous disease mutation in human iPS cells. Exp Hematol (2015) 0.90
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Advances in genetic therapeutic strategies for Duchenne muscular dystrophy. Exp Physiol (2015) 0.81
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CRISPR/Cas9 Flexes Its Muscles: In Vivo Somatic Gene Editing for Muscular Dystrophy. Mol Ther (2016) 0.79
Induced pluripotent stem cells for modeling neurological disorders. World J Transplant (2015) 0.79
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The Rise of CRISPR/Cas for Genome Editing in Stem Cells. Stem Cells Int (2016) 0.78
The use of stem cells to study autism spectrum disorder. Yale J Biol Med (2015) 0.78
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Homologous Recombination-Independent Large Gene Cassette Knock-in in CHO Cells Using TALEN and MMEJ-Directed Donor Plasmids. Int J Mol Sci (2015) 0.78
Minimizing off-Target Mutagenesis Risks Caused by Programmable Nucleases. Int J Mol Sci (2015) 0.77
The emerging role of viral vectors as vehicles for DMD gene editing. Genome Med (2016) 0.76
Enhanced Reprogramming Efficiency and Kinetics of Induced Pluripotent Stem Cells Derived from Human Duchenne Muscular Dystrophy. PLoS Curr (2015) 0.76
Direct reprogramming of urine-derived cells with inducible MyoD for modeling human muscle disease. Skelet Muscle (2016) 0.76
Concurrent progress of reprogramming and gene correction to overcome therapeutic limitation of mutant ALK2-iPSC. Exp Mol Med (2016) 0.76
The therapeutic potential of genome editing for β-thalassemia. F1000Res (2015) 0.76
Genome Editing Using Mammalian Haploid Cells. Int J Mol Sci (2015) 0.76
Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System. Mol Ther Nucleic Acids (2017) 0.76
Modern Genome Editing Technologies in Huntington's Disease Research. J Huntingtons Dis (2017) 0.75
CRISPR-Cas9 directed knock-out of a constitutively expressed gene using lance array nanoinjection. Springerplus (2016) 0.75
Spermatogonial stem cell autotransplantation and germline genomic editing: a future cure for spermatogenic failure and prevention of transmission of genomic diseases. Hum Reprod Update (2016) 0.75
Programmable Genome Editing Tools and their Regulation for Efficient Genome Engineering. Comput Struct Biotechnol J (2017) 0.75
Generation of skeletal muscle cells from pluripotent stem cells: advances and challenges. Front Cell Dev Biol (2015) 0.75
Aquapuncture Using Stem Cell Therapy to Treat Mdx Mice. Evid Based Complement Alternat Med (2015) 0.75
May I Cut in? Gene Editing Approaches in Human Induced Pluripotent Stem Cells. Cells (2017) 0.75
Memory or amnesia: the dilemma of stem cell therapy in muscular dystrophies. J Clin Invest (2015) 0.75
Advances in gene therapy for muscular dystrophies. F1000Res (2016) 0.75
SOX10-Nano-Lantern Reporter Human iPS Cells; A Versatile Tool for Neural Crest Research. PLoS One (2017) 0.75
An update on stem cell biology and engineering for brain development. Mol Psychiatry (2017) 0.75
Genome Editing Gene Therapy for Duchenne Muscular Dystrophy. J Neuromuscul Dis (2015) 0.75
Myogenic Differentiation from MYOGENIN-Mutated Human iPS Cells by CRISPR/Cas9. Stem Cells Int (2017) 0.75
Targeted RP9 ablation and mutagenesis in mouse photoreceptor cells by CRISPR-Cas9. Sci Rep (2017) 0.75
Adenoviral vectors encoding CRISPR/Cas9 multiplexes rescue dystrophin synthesis in unselected populations of DMD muscle cells. Sci Rep (2016) 0.75
Dystrophic Cardiomyopathy-Potential Role of Calcium in Pathogenesis, Treatment and Novel Therapies. Genes (Basel) (2017) 0.75
Young at Heart: Pioneering Approaches to Model Nonischaemic Cardiomyopathy with Induced Pluripotent Stem Cells. Stem Cells Int (2016) 0.75
Development of a Xeno-Free Feeder-Layer System from Human Umbilical Cord Mesenchymal Stem Cells for Prolonged Expansion of Human Induced Pluripotent Stem Cells in Culture. PLoS One (2016) 0.75
Can Human Pluripotent Stem Cell-Derived Cardiomyocytes Advance Understanding of Muscular Dystrophies? J Neuromuscul Dis (2016) 0.75
Calcium dysregulation contributes to neurodegeneration in FTLD patient iPSC-derived neurons. Sci Rep (2016) 0.75
Nat1 promotes translation of specific proteins that induce differentiation of mouse embryonic stem cells. Proc Natl Acad Sci U S A (2016) 0.75
CRISPR-Cas9: a promising tool for gene editing on induced pluripotent stem cells. Korean J Intern Med (2017) 0.75
Modeling craniofacial and skeletal congenital birth defects to advance therapies. Hum Mol Genet (2016) 0.75
Proteasome impairment in neural cells derived from HMSN-P patient iPSCs. Mol Brain (2017) 0.75
CRISPR/Cas9-Mediated Genome Editing Corrects Dystrophin Mutation in Skeletal Muscle Stem Cells in a Mouse Model of Muscle Dystrophy. Mol Ther Nucleic Acids (2017) 0.75
Circulating Biomarkers for Duchenne Muscular Dystrophy. J Neuromuscul Dis (2015) 0.75
Human stem cell modeling in neurofibromatosis type 1 (NF1). Exp Neurol (2017) 0.75
Genome engineering: a new approach to gene therapy for neuromuscular disorders. Nat Rev Neurol (2017) 0.75
Cellular Reprogramming, Genome Editing, and Alternative CRISPR Cas9 Technologies for Precise Gene Therapy of Duchenne Muscular Dystrophy. Stem Cells Int (2017) 0.75
Therapeutic opportunities and challenges of induced pluripotent stem cells-derived motor neurons for treatment of amyotrophic lateral sclerosis and motor neuron disease. Neural Regen Res (2017) 0.75
High-Throughput Imaging for the Discovery of Cellular Mechanisms of Disease. Trends Genet (2017) 0.75
Skeletal Muscle Cell Induction from Pluripotent Stem Cells. Stem Cells Int (2017) 0.75
Cell-type-specific genome editing with a microRNA-responsive CRISPR-Cas9 switch. Nucleic Acids Res (2017) 0.75
Rapid Generation of Human Genetic Loss-of-Function iPSC Lines by Simultaneous Reprogramming and Gene Editing. Stem Cell Reports (2017) 0.75
Characteristic analyses of a neural differentiation model from iPSC-derived neuron according to morphology, physiology, and global gene expression pattern. Sci Rep (2017) 0.75
Functional correction of dystrophin actin binding domain mutations by genome editing. JCI Insight (2017) 0.75
Genome editing: a robust technology for human stem cells. Cell Mol Life Sci (2017) 0.75
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Multiplex genome engineering using CRISPR/Cas systems. Science (2013) 55.53
RNA-guided human genome engineering via Cas9. Science (2013) 48.29
DNA targeting specificity of RNA-guided Cas9 nucleases. Nat Biotechnol (2013) 19.99
High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells. Nat Biotechnol (2013) 18.01
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Genetic engineering of human pluripotent cells using TALE nucleases. Nat Biotechnol (2011) 11.76
Searching for SNPs with cloud computing. Genome Biol (2009) 10.12
A more efficient method to generate integration-free human iPS cells. Nat Methods (2011) 7.39
Dynamic changes in the copy number of pluripotency and cell proliferation genes in human ESCs and iPSCs during reprogramming and time in culture. Cell Stem Cell (2011) 6.82
Generation of isogenic pluripotent stem cells differing exclusively at two early onset Parkinson point mutations. Cell (2011) 6.56
A TALEN genome-editing system for generating human stem cell-based disease models. Cell Stem Cell (2012) 4.49
Dystrophin immunity in Duchenne's muscular dystrophy. N Engl J Med (2010) 4.28
Enhanced efficiency of human pluripotent stem cell genome editing through replacing TALENs with CRISPRs. Cell Stem Cell (2013) 4.05
Screening ethnically diverse human embryonic stem cells identifies a chromosome 20 minimal amplicon conferring growth advantage. Nat Biotechnol (2011) 3.97
Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease. Blood (2011) 3.43
Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease-mediated safe harbor targeting. Blood (2011) 3.21
Low incidence of off-target mutations in individual CRISPR-Cas9 and TALEN targeted human stem cell clones detected by whole-genome sequencing. Cell Stem Cell (2014) 3.16
Genotype-phenotype analysis in 2,405 patients with a dystrophinopathy using the UMD-DMD database: a model of nationwide knowledgebase. Hum Mutat (2009) 2.93
E-CRISP: fast CRISPR target site identification. Nat Methods (2014) 2.73
Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations. Hum Mutat (2009) 2.67
CRISPR/Cas9 systems have off-target activity with insertions or deletions between target DNA and guide RNA sequences. Nucleic Acids Res (2014) 2.59
Whole-genome sequencing analysis reveals high specificity of CRISPR/Cas9 and TALEN-based genome editing in human iPSCs. Cell Stem Cell (2014) 2.57
Cas-OFFinder: a fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases. Bioinformatics (2014) 2.54
In situ genetic correction of the sickle cell anemia mutation in human induced pluripotent stem cells using engineered zinc finger nucleases. Stem Cells (2011) 2.52
Isolation of human iPS cells using EOS lentiviral vectors to select for pluripotency. Nat Methods (2009) 2.51
Targeted gene correction minimally impacts whole-genome mutational load in human-disease-specific induced pluripotent stem cell clones. Cell Stem Cell (2014) 2.24
Characterization of genomic deletion efficiency mediated by clustered regularly interspaced palindromic repeats (CRISPR)/Cas9 nuclease system in mammalian cells. J Biol Chem (2014) 2.18
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD. Mol Ther (2011) 2.06
Human ES- and iPS-derived myogenic progenitors restore DYSTROPHIN and improve contractility upon transplantation in dystrophic mice. Cell Stem Cell (2012) 2.06
Efficient TALEN construction and evaluation methods for human cell and animal applications. Genes Cells (2013) 1.99
TALEN-based gene correction for epidermolysis bullosa. Mol Ther (2013) 1.84
Microhomology-based choice of Cas9 nuclease target sites. Nat Methods (2014) 1.82
The uniqueome: a mappability resource for short-tag sequencing. Bioinformatics (2010) 1.79
Repeating pattern of non-RVD variations in DNA-binding modules enhances TALEN activity. Sci Rep (2013) 1.73
Reading frame correction by targeted genome editing restores dystrophin expression in cells from Duchenne muscular dystrophy patients. Mol Ther (2013) 1.63
Seamless modification of wild-type induced pluripotent stem cells to the natural CCR5Δ32 mutation confers resistance to HIV infection. Proc Natl Acad Sci U S A (2014) 1.49
Efficient and reproducible myogenic differentiation from human iPS cells: prospects for modeling Miyoshi Myopathy in vitro. PLoS One (2013) 1.34
Efficient drug screening and gene correction for treating liver disease using patient-specific stem cells. Hepatology (2013) 1.34
Myoblasts derived from normal hESCs and dystrophic hiPSCs efficiently fuse with existing muscle fibers following transplantation. Mol Ther (2012) 1.33
An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cells. Nat Commun (2013) 1.17
Transcription activator-like effector nuclease (TALEN)-mediated gene correction in integration-free β-thalassemia induced pluripotent stem cells. J Biol Chem (2013) 1.13
Genetic and chemical correction of cholesterol accumulation and impaired autophagy in hepatic and neural cells derived from Niemann-Pick Type C patient-specific iPS cells. Stem Cell Reports (2014) 1.11
Gene correction of a duchenne muscular dystrophy mutation by meganuclease-enhanced exon knock-in. Hum Gene Ther (2013) 1.08
Genetic correction using engineered nucleases for gene therapy applications. Dev Growth Differ (2013) 0.93
Endonucleases: tools to correct the dystrophin gene. J Gene Med (2011) 0.92
Delivery of full-length factor VIII using a piggyBac transposon vector to correct a mouse model of hemophilia A. PLoS One (2014) 0.89
Induced pluripotent stem cell generation-associated point mutations arise during the initial stages of the conversion of these cells. Stem Cell Reports (2014) 0.89
Current Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy. Pharmaceuticals (Basel) (2013) 0.82
Repeating pattern of non-RVD variations in DNA-binding modules enhances TALEN activity. Sci Rep (2013) 1.73
Simple knockout by electroporation of engineered endonucleases into intact rat embryos. Sci Rep (2014) 1.32
The Microtubule-Depolymerizing Activity of a Mitotic Kinesin Protein KIF2A Drives Primary Cilia Disassembly Coupled with Cell Proliferation. Cell Rep (2015) 0.91
Stochastic promoter activation affects Nanog expression variability in mouse embryonic stem cells. Sci Rep (2014) 0.87
Forcible destruction of severely misfolded mammalian glycoproteins by the non-glycoprotein ERAD pathway. J Cell Biol (2015) 0.83
Down syndrome-associated haematopoiesis abnormalities created by chromosome transfer and genome editing technologies. Sci Rep (2014) 0.81
Smarcal1 promotes double-strand-break repair by nonhomologous end-joining. Nucleic Acids Res (2015) 0.80
Genome Editing in Mouse Spermatogonial Stem Cell Lines Using TALEN and Double-Nicking CRISPR/Cas9. Stem Cell Reports (2015) 0.78
Base-Resolution Methylome of Retinal Pigment Epithelial Cells Used in the First Trial of Human Induced Pluripotent Stem Cell-Based Autologous Transplantation. Stem Cell Reports (2019) 0.75
PLK1-mediated phosphorylation of WDR62/MCPH2 ensures proper mitotic spindle orientation. Hum Mol Genet (2017) 0.75