New developments in the use of gene therapy to treat Duchenne muscular dystrophy

Expert Opinion on Biological Therapy

Volumn 14, Issue 2, 2014, Pages 209-230

  Jarmin, Susan ^a   Kymalainen, Hanna ^a   Popplewell, Linda ^a   Dickson, George ^a  

^a UNIVERSITY OF LONDON   (United Kingdom)

Author keywords

Exon skipping; Gene editing; Gene therapy; Muscular dystrophy

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DYSTROPHIN; OLIGONUCLEOTIDE;

ADENO ASSOCIATED VIRUS; CONJUGATION; DUCHENNE MUSCULAR DYSTROPHY; EXON SKIPPING; GENE MUTATION; GENE TARGETING; GENETIC ENHANCEMENT; GENOME; HEART MUSCLE; HUMAN; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; NONVIRAL GENE THERAPY; OPEN READING FRAME; PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PHENOTYPE; REVIEW; SKELETAL MUSCLE; SPLICEOSOME; STOP CODON; TARGETED GENE REPAIR; TRANS SPLICING; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRAL TROPISM;

ANIMALS; CLINICAL TRIALS, PHASE II AS TOPIC; CLINICAL TRIALS, PHASE III AS TOPIC; CODON, TERMINATOR; DEPENDOVIRUS; DYSTROPHIN; EXONS; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MICE; MUSCULAR DYSTROPHY, DUCHENNE; MUTATION; OLIGONUCLEOTIDES, ANTISENSE; OPEN READING FRAMES; PHENOTYPE; RNA SPLICING; TRANSGENES;

EID: 84891922503     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712598.2014.866087     Document Type: Review

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