Repair or replace? Exploiting novel gene and cell therapy strategies for muscular dystrophies

FEBS Journal

Volumn 280, Issue 17, 2013, Pages 4263-4280

  Benedetti, Sara ^a   Hoshiya, Hidetoshi ^a   Tedesco, Francesco Saverio ^a,b  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

^b SAN RAFFAELE HOSPITAL   (Italy)

Author keywords

cell therapy; gene therapy; genome editing; mRNA modifications; muscle regeneration; muscular dystrophy; stem cells

Indexed keywords

ADENOVIRUS VECTOR; ATALUREN; CD133 ANTIGEN; DRISAPERSEN; ENDONUCLEASE; ETEPLIRSEN; GENTAMICIN; HERPES SIMPLEX VECTOR; LENTIVIRUS VECTOR; PARVOVIRUS VECTOR; PLACEBO; PLASMID VECTOR; SMALL NUCLEAR RNA; TRANSPOSON RESOLVASE; UNCLASSIFIED DRUG; VIRUS VECTOR;

ALLOGENEIC STEM CELL TRANSPLANTATION; ANTISENSE THERAPY; ARTIFICIAL CHROMOSOME; BECKER MUSCULAR DYSTROPHY; CD133+ STEM CELL; CELL THERAPY; CLONING VECTOR; DISEASE SEVERITY; DUCHENNE MUSCULAR DYSTROPHY; EMBRYONIC STEM CELL; EXON SKIPPING; GENE EXPRESSION; GENE MUTATION; GENE REPLACEMENT THERAPY; GENE TARGETING; GENE THERAPY; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN ARTIFICIAL CHROMOSOME; MESENCHYMAL STEM CELL; MESOANGIOBLAST; MUSCLE DERIVED STEM CELL; MUSCULAR DYSTROPHY; MYOBLAST; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PERICYTE; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PLURIPOTENT STEM CELL; PRIORITY JOURNAL; PW1 INTERSTITIAL CELL; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; SATELLITE CELL; SKELETAL MUSCLE; TRANSPOSON; VIRAL GENE DELIVERY SYSTEM;

CELL THERAPY; GENE THERAPY; GENOME EDITING; MRNA MODIFICATIONS; MUSCLE REGENERATION; MUSCULAR DYSTROPHY; STEM CELLS;

ANIMALS; GENETIC THERAPY; HUMANS; MUSCULAR DYSTROPHY, DUCHENNE; STEM CELL TRANSPLANTATION; STEM CELLS; TISSUE THERAPY;

EID: 84882656215     PISSN: 1742464X     EISSN: 17424658     Source Type: Journal    
DOI: 10.1111/febs.12178     Document Type: Review

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