| scholarly article | Q13442814 |
| review article | Q7318358 |
| P50 | author | Akitsu Hotta | Q55135226 |
| P2093 | author name string | Akitsu Hotta | |
| P2860 | cites work | Transplantation of retroviral producer cells for in vivo gene transfer into mouse skeletal muscle | Q71624177 |
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| A highly stable and nonintegrated human artificial chromosome (HAC) containing the 2.4 Mb entire human dystrophin gene | Q24631410 | ||
| Disease-specific induced pluripotent stem cells | Q24641981 | ||
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| A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity | Q24669850 | ||
| Efficient and reproducible myogenic differentiation from human iPS cells: prospects for modeling Miyoshi Myopathy in vitro | Q27324593 | ||
| Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors | Q27860937 | ||
| Induction of pluripotent stem cells from adult human fibroblasts by defined factors | Q27860967 | ||
| Zscan4 regulates telomere elongation and genomic stability in ES cells | Q28116840 | ||
| Revealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection | Q28245039 | ||
| The CRISPR/Cas bacterial immune system cleaves bacteriophage and plasmid DNA | Q28297640 | ||
| CRISPR--a widespread system that provides acquired resistance against phages in bacteria and archaea | Q29614740 | ||
| CRISPR interference limits horizontal gene transfer in staphylococci by targeting DNA | Q29615786 | ||
| Identification of genes that are associated with DNA repeats in prokaryotes | Q29615790 | ||
| DNA targeting specificity of RNA-guided Cas9 nucleases | Q29615793 | ||
| Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting | Q29616145 | ||
| Molecular reconstruction of Sleeping Beauty, a Tc1-like transposon from fish, and its transposition in human cells | Q29617246 | ||
| Dystrophin: the protein product of the Duchenne muscular dystrophy locus | Q29618077 | ||
| rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice. | Q30602105 | ||
| Dystrophin delivery to muscles of mdx mice using lentiviral vectors leads to myogenic progenitor targeting and stable gene expression. | Q33730444 | ||
| CRISPR/Cas9 systems have off-target activity with insertions or deletions between target DNA and guide RNA sequences | Q33791286 | ||
| Low incidence of off-target mutations in individual CRISPR-Cas9 and TALEN targeted human stem cell clones detected by whole-genome sequencing. | Q33849553 | ||
| DNA interrogation by the CRISPR RNA-guided endonuclease Cas9. | Q33931841 | ||
| In vivo genome editing using Staphylococcus aureus Cas9 | Q34043628 | ||
| Meganucleases can restore the reading frame of a mutated dystrophin | Q34110253 | ||
| Function and genetics of dystrophin and dystrophin-related proteins in muscle | Q34120764 | ||
| Systemic administration of PRO051 in Duchenne's muscular dystrophy | Q34172661 | ||
| Microhomology-based choice of Cas9 nuclease target sites. | Q53515678 | ||
| Dystrophin expression in muscles of duchenne muscular dystrophy patients after high-density injections of normal myogenic cells. | Q54602083 | ||
| Immortalized Skin Fibroblasts Expressing Conditional MyoD as a Renewable and Reliable Source of Converted Human Muscle Cells to Assess Therapeutic Strategies for Muscular Dystrophies: Validation of an Exon-Skipping Approach to Restore Dystrophin in D | Q57268497 | ||
| Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene | Q59053234 | ||
| Transduction of Full-length Dystrophin to Multiple Skeletal Muscles Improves Motor Performance and Life Span in Utrophin/Dystrophin Double Knockout Mice | Q59876164 | ||
| Becker muscular dystrophy severity is linked to the structure of dystrophin | Q63192023 | ||
| Isolation of candidate cDNAs for portions of the Duchenne muscular dystrophy gene | Q34190227 | ||
| Functional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin. | Q34190967 | ||
| Advances in Duchenne muscular dystrophy gene therapy | Q34267400 | ||
| Systemic delivery of genes to striated muscles using adeno-associated viral vectors. | Q34347199 | ||
| Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA | Q34433586 | ||
| Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy. | Q34463483 | ||
| Expression of full-length utrophin prevents muscular dystrophy in mdx mice | Q34482808 | ||
| Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule | Q34537558 | ||
| Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection | Q34770058 | ||
| Adeno-associated viral (AAV) vectors do not efficiently target muscle satellite cells | Q34886209 | ||
| Gene therapy of muscular dystrophy | Q34915108 | ||
| Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations | Q34926082 | ||
| Current status of pharmaceutical and genetic therapeutic approaches to treat DMD. | Q34994545 | ||
| Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8. | Q35025591 | ||
| Genome-wide recessive genetic screening in mammalian cells with a lentiviral CRISPR-guide RNA library | Q35096218 | ||
| Correction of dystrophin expression in cells from Duchenne muscular dystrophy patients through genomic excision of exon 51 by zinc finger nucleases | Q35153108 | ||
| Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model | Q35573761 | ||
| Human ES- and iPS-derived myogenic progenitors restore DYSTROPHIN and improve contractility upon transplantation in dystrophic mice. | Q35947481 | ||
| Mutational spectrum of DMD mutations in dystrophinopathy patients: application of modern diagnostic techniques to a large cohort | Q36116293 | ||
| Filia Is an ESC-Specific Regulator of DNA Damage Response and Safeguards Genomic Stability | Q36176638 | ||
| Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery | Q36187202 | ||
| FLASH assembly of TALENs for high-throughput genome editing | Q36573684 | ||
| Characteristics of Japanese Duchenne and Becker muscular dystrophy patients in a novel Japanese national registry of muscular dystrophy (Remudy). | Q36803129 | ||
| Development of multiexon skipping antisense oligonucleotide therapy for Duchenne muscular dystrophy | Q37105210 | ||
| Reading frame correction by targeted genome editing restores dystrophin expression in cells from Duchenne muscular dystrophy patients. | Q37181372 | ||
| Retroviral vector silencing during iPS cell induction: an epigenetic beacon that signals distinct pluripotent states | Q37261459 | ||
| CRISPR/Cas9 systems targeting β-globin and CCR5 genes have substantial off-target activity | Q37271186 | ||
| Repeating pattern of non-RVD variations in DNA-binding modules enhances TALEN activity. | Q37349204 | ||
| Genetic correction of dystrophin deficiency and skeletal muscle remodeling in adult MDX mouse via transplantation of retroviral producer cells. | Q37370555 | ||
| Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases. | Q37420333 | ||
| Enhanced efficiency of human pluripotent stem cell genome editing through replacing TALENs with CRISPRs | Q37585983 | ||
| Genetic therapeutic approaches for Duchenne muscular dystrophy | Q38014583 | ||
| Relevance of sequence and structure elements for deletion events in the dystrophin gene major hot-spot | Q38357429 | ||
| Whole-genome sequencing analysis reveals high specificity of CRISPR/Cas9 and TALEN-based genome editing in human iPSCs | Q38977941 | ||
| An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cells | Q39456951 | ||
| Endonucleases: tools to correct the dystrophin gene. | Q39466873 | ||
| Complete genetic correction of ips cells from Duchenne muscular dystrophy | Q39641162 | ||
| Immortalized pathological human myoblasts: towards a universal tool for the study of neuromuscular disorders | Q39738924 | ||
| Optimization of peptide nucleic acid antisense oligonucleotides for local and systemic dystrophin splice correction in the mdx mouse. | Q39882118 | ||
| Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypes | Q40574948 | ||
| Herpes simplex virus vector-mediated dystrophin gene transfer and expression in MDX mouse skeletal muscle | Q40890814 | ||
| Effective restoration of dystrophin-associated proteins in vivo by adenovirus-mediated transfer of truncated dystrophin cDNAs | Q41048543 | ||
| Myoblasts derived from normal hESCs and dystrophic hiPSCs efficiently fuse with existing muscle fibers following transplantation | Q41136531 | ||
| Gene transfer to muscle using herpes simplex virus-based vectors | Q41575051 | ||
| Dystrophin immunity in Duchenne's muscular dystrophy. | Q41590387 | ||
| Derivation and expansion of PAX7-positive muscle progenitors from human and mouse embryonic stem cells | Q41823910 | ||
| Precise correction of the dystrophin gene in duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9. | Q41873090 | ||
| A zebrafish embryo culture system defines factors that promote vertebrate myogenesis across species. | Q41892400 | ||
| Gene correction of a duchenne muscular dystrophy mutation by meganuclease-enhanced exon knock-in. | Q42289009 | ||
| Targeted gene disruption in somatic zebrafish cells using engineered TALENs | Q42862072 | ||
| Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice | Q44264343 | ||
| Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches | Q44671291 | ||
| In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping | Q45002735 | ||
| Adeno-associated virus vector gene transfer and sarcolemmal expression of a 144 kDa micro-dystrophin effectively restores the dystrophin-associated protein complex and inhibits myofibre degeneration in nude/mdx mice | Q45733070 | ||
| Stable transduction of myogenic cells with lentiviral vectors expressing a minidystrophin | Q45881869 | ||
| Stable micro-dystrophin gene transfer using an integrating adeno-retroviral hybrid vector ameliorates the dystrophic pathology in mdx mouse muscle. | Q45887463 | ||
| Genotype-phenotype analysis in 2,405 patients with a dystrophinopathy using the UMD-DMD database: a model of nationwide knowledgebase | Q48006442 | ||
| P275 | copyright license | Creative Commons Attribution-NonCommercial 4.0 International | Q34179348 |
| P6216 | copyright status | copyrighted | Q50423863 |
| P433 | issue | 4 | |
| P921 | main subject | gene therapy | Q213901 |
| P304 | page(s) | 343-355 | |
| P577 | publication date | 2015-09-01 | |
| P1433 | published in | Journal of neuromuscular diseases | Q27726242 |
| P1476 | title | Genome Editing Gene Therapy for Duchenne Muscular Dystrophy | |
| P478 | volume | 2 |
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