Ex vivo gene editing of the dystrophin gene in muscle stem cells mediated by peptide nucleic acid single stranded oligodeoxynucleotides induces stable expression of dystrophin in a mouse model for Duchenne muscular dystrophy

Stem Cells

Volumn 32, Issue 7, 2014, Pages 1817-1830

  Nik Ahd, Farnoosh ^a   Bertoni, Carmen ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

Duchenne muscular dystrophy; Dystrophin; Gene editing; Gene repair; Muscle progenitor cells; Muscle stem cell; Satellite cells; Single stranded oligodeoxynucleotides

Indexed keywords

DYSTROPHIN; GENOMIC DNA; OLIGODEOXYNUCLEOTIDE; PEPTIDE NUCLEIC ACID;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DUCHENNE MUSCULAR DYSTROPHY; EX VIVO STUDY; IMMUNOFLUORESCENCE; MOUSE; MUSCLE CELL; NONHUMAN; PROTEIN EXPRESSION; SELF CONCEPT; SKELETAL MUSCLE; STABLE EXPRESSION; STEM CELL; ANIMAL; C57BL MOUSE; CELL CULTURE; GENE EXPRESSION; GENE THERAPY; GENETIC ENGINEERING; GENETIC TRANSFECTION; GENETICS; HUMAN; METABOLISM; MYOBLAST; NUDE MOUSE; PATHOLOGY; REGENERATIVE MEDICINE; TRANSPLANTATION; X CHROMOSOME LINKED MUSCULAR DYSTROPHIC MOUSE;

ANIMALS; CELLS, CULTURED; DYSTROPHIN; GENE EXPRESSION; GENETIC ENGINEERING; GENETIC THERAPY; HUMANS; MICE, INBRED C57BL; MICE, INBRED MDX; MICE, NUDE; MUSCLE FIBERS, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE; MYOBLASTS, SKELETAL; PEPTIDE NUCLEIC ACIDS; REGENERATIVE MEDICINE; TRANSFECTION;

EID: 84901025931     PISSN: 10665099     EISSN: 15494918     Source Type: Journal    
DOI: 10.1002/stem.1668     Document Type: Article

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