Full-length Dysferlin Transfer by the Hyperactive Sleeping Beauty Transposase Restores Dysferlin-deficient Muscle

Molecular Therapy Nucleic Acids

Volumn 5, Issue , 2016, Pages e277-

  Escobar, Helena ^a   Schöwel, Verena ^a   Spuler, Simone ^a   Marg, Andreas ^a   Izsvák, Zsuzsanna ^a  

^a MAX DELBRÜCK CENTER FOR MOLECULAR MEDICINE   (Germany)

Author keywords

dysferlin; gene therapy; myoblast transplantation; Sleeping Beauty transposon

Indexed keywords

CARDIOTOXIN; COMPLEMENTARY DNA; DYSFERLIN; PLASMID DNA; SLEEPING BEAUTY TRANSPOSASE; TRANSCRIPTION FACTOR PAX7; TRANSPOSASE; UNCLASSIFIED DRUG;

ALLELE; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL DIFFERENTIATION; CELL ENGINEERING; CONTROLLED STUDY; ENGRAFTMENT; EXPRESSION VECTOR; FEMALE; FLUORESCENCE ACTIVATED CELL SORTING; GENE EXPRESSION; GENE MUTATION; HUMAN; HUMAN CELL; IMMUNE DEFICIENCY; IMMUNOHISTOCHEMISTRY; INTERNAL RIBOSOME ENTRY SITE; IRRADIATION; MOUSE; MUSCLE CELL; MUSCLE REGENERATION; MYOBLAST; MYOTUBE; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PRIORITY JOURNAL; REGULATORY SEQUENCE; SARCOLEMMA; SATELLITE CELL; SKELETAL MUSCLE; TIBIALIS ANTERIOR MUSCLE; TRANSPOSON;

EID: 85015432307     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1038/mtna.2015.52     Document Type: Article

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