A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells - Wikidata - awgldk - TriplyDB

A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells

A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells

http://www.wikidata.org/entity/Q36782735

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Empower multiplex cell and tissue-specific CRISPR-mediated gene manipulation with self-cleaving ribozymes and tRNA.CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice.Genome Editing and Muscle Stem Cells as a Therapeutic Tool for Muscular Dystrophies.Skeletal muscle generated from induced pluripotent stem cells - induction and application.Expansion and Purification Are Critical for the Therapeutic Application of Pluripotent Stem Cell-Derived Myogenic Progenitors.Adenoviral vectors encoding CRISPR/Cas9 multiplexes rescue dystrophin synthesis in unselected populations of DMD muscle cells.Induced Pluripotent Stem Cells Meet Genome Editing Restoring Ureagenesis in Hepatocytes by CRISPR/Cas9-mediated Genomic Addition to Arginase-deficient Induced Pluripotent Stem Cells.Genome-wide Specificity of Highly Efficient TALENs and CRISPR/Cas9 for T Cell Receptor Modification.CRISPR/Cas9-Mediated Genome Editing Corrects Dystrophin Mutation in Skeletal Muscle Stem Cells in a Mouse Model of Muscle Dystrophy.Generation of skeletal myogenic progenitors from human pluripotent stem cells using non-viral delivery of minicircle DNA.Editing the genome of hiPSC with CRISPR/Cas9: disease models.Generation of human muscle fibers and satellite-like cells from human pluripotent stem cells in vitro.Exon skipping: a first in class strategy for Duchenne muscular dystrophy.Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy.Gene correction in patient-specific iPSCs for therapy development and disease modeling.Genome editing comes of age.Therapeutic applications of CRISPR RNA-guided genome editing.Genome Editing of Monogenic Neuromuscular Diseases: A Systematic Review.Modeling of Autism Using Organoid Technology.Human iPSC-derived cardiomyocytes and tissue engineering strategies for disease modeling and drug screening.Intersections of post-transcriptional gene regulatory mechanisms with intermediary metabolism.Cell therapy for GI motility disorders: comparison of cell sources and proposed steps for treating Hirschsprung disease.Therapeutic gene editing: delivery and regulatory perspectives Progress toward Gene Therapy for Duchenne Muscular Dystrophy.Cellular Reprogramming, Genome Editing, and Alternative CRISPR Cas9 Technologies for Precise Gene Therapy of Duchenne Muscular Dystrophy.Gene editing advance re-ignites debate on the merits and risks of animal to human transplantation.Creation of a Novel Humanized Dystrophic Mouse Model of Duchenne Muscular Dystrophy and Application of a CRISPR/Cas9 Gene Editing Therapy.Genome engineering: a new approach to gene therapy for neuromuscular disorders.Functional correction of dystrophin actin binding domain mutations by genome editing.In Vivo Human Somitogenesis Guides Somite Development from hPSCs.Drusen in patient-derived hiPSC-RPE models of macular dystrophies.Next-generation mapping: a novel approach for detection of pathogenic structural variants with a potential utility in clinical diagnosis.Lectin-binding characterizes the healthy human skeletal muscle glycophenotype and identifies disease-specific changes in dystrophic muscle.Recent developments in Duchenne muscular dystrophy: facts and numbers.Viral Vectors, Engineered Cells and the CRISPR Revolution.Reversible immortalisation enables genetic correction of human muscle progenitors and engineering of next-generation human artificial chromosomes for Duchenne muscular dystrophy.Engineering micromyocardium to delineate cellular and extracellular regulation of myocardial tissue contractility.Characterization of Gene Alterations following Editing of the β-Globin Gene Locus in Hematopoietic Stem/Progenitor Cells.Glucose inhibits cardiac muscle maturation through nucleotide biosynthesis.

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A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells

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A Single CRISPR-Cas9 Deletion ...... in hiPSC-Derived Muscle Cells

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J.STEM.2016.01.021

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A Single CRISPR-Cas9 Deletion ...... in hiPSC-Derived Muscle Cells

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April D Pyle

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Atsushi Nakano

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Chino Kumagai-Cresse

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Courtney S Young

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Derek Wang

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Donald B Kohn

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Haruko Nakano

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Jerome A Zack

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M Carrie Miceli

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Majib Jan

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P2860

miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy

Clinical features of patients with dystrophinopathy sharing the 45-55 exon deletion of DMD gene

Dystrophin-deficient cardiomyocytes derived from human urine: new biologic reagents for drug discovery.

CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice

Spell Checking Nature: Versatility of CRISPR/Cas9 for Developing Treatments for Inherited Disorders.

Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA

Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy.

COSMID: A Web-based Tool for Identifying and Validating CRISPR/Cas Off-target Sites

Myoblast transplantation.

Long-term efficacy of systemic multiexon skipping targeting dystrophin exons 45-55 with a cocktail of vivo-morpholinos in mdx52 mice

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