Found: 28
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Minimizing off-Target Mutagenesis Risks Caused by Programmable Nucleases.
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- International Journal of Molecular Sciences, 2015, v. 16, n. 10, p. 24751, doi. 10.3390/ijms161024751
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- Article
Genetic correction using engineered nucleases for gene therapy applications.
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- Development, Growth & Differentiation, 2014, v. 56, n. 1, p. 63, doi. 10.1111/dgd.12107
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Establishment of quantitative and consistent in vitro skeletal muscle pathological models of myotonic dystrophy type 1 using patient-derived iPSCs.
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- Scientific Reports, 2023, v. 13, n. 1, p. 1, doi. 10.1038/s41598-022-26614-z
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- Article
Cell-type-specific genome editing with a microRNA-responsive CRISPR-Cas9 switch.
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- Nucleic Acids Research, 2017, v. 45, n. 13, p. 1, doi. 10.1093/nar/gkx309
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- Article
Low immunogenicity of LNP allows repeated administrations of CRISPR-Cas9 mRNA into skeletal muscle in mice.
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- Nature Communications, 2021, v. 12, n. 1, p. 1, doi. 10.1038/s41467-021-26714-w
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- Article
Isolation of human iPS cells using EOS lentiviral vectors to select for pluripotency.
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- Nature Methods, 2009, v. 6, n. 5, p. 370, doi. 10.1038/nmeth.1325
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- Article
Calcium dysregulation contributes to neurodegeneration in FTLD patient iPSC-derived neurons.
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- Scientific Reports, 2016, p. 34904, doi. 10.1038/srep34904
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- Article
Delivery of Full-Length Factor VIII Using a <i>piggyBac</i> Transposon Vector to Correct a Mouse Model of Hemophilia A.
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- PLoS ONE, 2014, v. 9, n. 8, p. 1, doi. 10.1371/journal.pone.0104957
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Efficient and Reproducible Myogenic Differentiation from Human iPS Cells: Prospects for Modeling Miyoshi Myopathy <i>In Vitro</i>.
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- PLoS ONE, 2013, v. 8, n. 4, p. 1, doi. 10.1371/journal.pone.0061540
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- Article
MECP2 Isoform-Specific Vectors with Regulated Expression for Rett Syndrome Gene Therapy.
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- PLoS ONE, 2009, v. 4, n. 8, p. 1, doi. 10.1371/journal.pone.0006810
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Retroviral vector silencing during iPS cell induction: An epigenetic beacon that signals distinct pluripotent states.
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- Journal of Cellular Biochemistry, 2008, v. 105, n. 4, p. 940, doi. 10.1002/jcb.21912
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CRISPR-Cas3 induces broad and unidirectional genome editing in human cells.
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- Nature Communications, 2019, v. 10, n. 1, p. 1, doi. 10.1038/s41467-019-13226-x
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- Article
CRISPR-Cas3 induces broad and unidirectional genome editing in human cells.
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- Nature Communications, 2019, p. 1, doi. 10.1038/s41467-019-13226-x
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- Article
Constitutive heterochromatin reorganization during somatic cell reprogramming.
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- EMBO Journal, 2011, v. 30, n. 9, p. 1778, doi. 10.1038/emboj.2011.96
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Cellular Reprogramming, Genome Editing, and Alternative CRISPR Cas9 Technologies for Precise Gene Therapy of Duchenne Muscular Dystrophy.
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- Stem Cells International, 2017, p. 1, doi. 10.1155/2017/8765154
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- Article
The Src/c-Abl pathway is a potential therapeutic target in amyotrophic lateral sclerosis.
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- Science Translational Medicine, 2017, v. 9, n. 391, p. 1, doi. 10.1126/scitranslmed.aaf3962
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- Article
A patient-derived iPSC model revealed oxidative stress increases facioscapulohumeral muscular dystrophy-causative DUX4.
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- Human Molecular Genetics, 2018, v. 27, n. 23, p. 4024, doi. 10.1093/hmg/ddy293
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Isolation of MECP2-null Rett Syndrome patient hiPS cells and isogenic controls through X-chromosome inactivation.
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- Human Molecular Genetics, 2011, v. 20, n. 11, p. 2103, doi. 10.1093/hmg/ddr093
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- Article
Cartilage tissue engineering identifies abnormal human induced pluripotent stem cells.
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- Scientific Reports, 2013, p. 1, doi. 10.1038/srep01978
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Contractile Activity of Myotubes Derived from Human Induced Pluripotent Stem Cells: A Model of Duchenne Muscular Dystrophy.
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- Cells (2073-4409), 2021, v. 10, n. 10, p. 2556, doi. 10.3390/cells10102556
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- Article
Srf destabilizes cellular identity by suppressing celltype-specific gene expression programs.
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- Nature Communications, 2018, v. 9, n. 1, p. 1, doi. 10.1038/s41467-018-03748-1
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Transplantation of neurons derived from human iPS cells cultured on collagen matrix into guinea-pig cochleae.
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- Journal of Tissue Engineering & Regenerative Medicine, 2017, v. 11, n. 6, p. 1766, doi. 10.1002/term.2072
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- Article
MiR-33a is a therapeutic target in SPG4-related hereditary spastic paraplegia human neurons.
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- Clinical Science, 2019, v. 133, n. 4, p. 583, doi. 10.1042/CS20180980
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β-Globin LCR and Intron Elements Cooperate and Direct Spatial Reorganization for Gene Therapy.
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- PLoS Genetics, 2008, v. 4, n. 4, p. 1, doi. 10.1371/journal.pgen.1000051
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CD151 expression marks atrial- and ventricular- differentiation from human induced pluripotent stem cells.
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- Communications Biology, 2024, v. 7, n. 1, p. 1, doi. 10.1038/s42003-024-05809-2
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- Article
iPSC-derived type IV collagen α5-expressing kidney organoids model Alport syndrome.
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- Communications Biology, 2023, v. 6, n. 1, p. 1, doi. 10.1038/s42003-023-05203-4
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- Article
Reprogramming progeria fibroblasts re-establishes a normal epigenetic landscape.
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- Aging Cell, 2017, v. 16, n. 4, p. 870, doi. 10.1111/acel.12621
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iMSC-mediated delivery of ACVR2B-Fc fusion protein reduces heterotopic ossification in a mouse model of fibrodysplasia ossificans progressiva.
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- Stem Cell Research & Therapy, 2024, v. 15, n. 1, p. 1, doi. 10.1186/s13287-024-03691-7
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- Article