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Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy

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AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9

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Serum glutamate dehydrogenase activity enables early detection of liver injury in subjects with underlying muscle impairments

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CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells

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A biodegradable nanocapsule delivers a Cas9 ribonucleoprotein complex for in vivo genome editing

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High levels of AAV vector integration into CRISPR-induced DNA breaks

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Functional Rescue of Dystrophin Deficiency in Mice Caused by Frameshift Mutations Using Campylobacter jejuni Cas9

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Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

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A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing

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Nanoparticle delivery of CRISPR into the brain rescues a mouse model of fragile X syndrome from exaggerated repetitive behaviours

(2018) Bumwhee Lee et al.   Nature Biomedical Engineering

CRISPR-Induced Deletion with SaCas9 Restores Dystrophin Expression in Dystrophic Models In Vitro and In Vivo

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In vivo CRISPR editing with no detectable genome-wide off-target mutations

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Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy

(2018) Leonela Amoasii et al.   SCIENCE

CRISPR Correction of Duchenne Muscular Dystrophy

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Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing

(2017) Hao Yin et al.   NATURE BIOTECHNOLOGY

CIRCLE-seq: a highly sensitive in vitro screen for genome-wide CRISPR–Cas9 nuclease off-targets

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

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Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair

(2017) Kunwoo Lee et al.   Nature Biomedical Engineering

Non-Viral CRISPR/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co-Delivery of Cas9 mRNA and sgRNA

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Don’t edit the human germ line

(2015) Edward Lanphier et al.   NATURE

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

(2015) C. Long et al.   SCIENCE

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

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In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

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Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9

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Polyplex nanomicelle promotes hydrodynamic gene introduction to skeletal muscle

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