New developments in exon skipping and splice modulation therapies for neuromuscular diseases

Expert Opinion on Biological Therapy

Volumn 14, Issue 6, 2014, Pages 809-819

  Touznik, Aleksander ^a   Lee, Joshua J A ^a   Yokota, Toshifumi ^a  

^a UNIVERSITY OF ALBERTA   (Canada)

Author keywords

Antisense therapy; Duchenne muscular dystrophy; Dysferlinopathy; Exon skipping; Fukuyama congenital muscular dystrophy; Limb girdle muscular dystrophy 2B; Miyoshi myopathy; Spinal muscular atrophy; Splice modulation

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; B 30; DRISAPERSEN; DYSFERLIN; DYSTROPHIN; ETEPLIRSEN; UNCLASSIFIED DRUG;

CELL SURVIVAL; DUCHENNE MUSCULAR DYSTROPHY; DYSFERLINOPATHY; EXON; FUKUYAMA CONGENITAL MUSCULAR DYSTROPHY; HUMAN; MOTONEURON; NEUROMUSCULAR DISEASE; REVIEW; SPINAL MUSCULAR ATROPHY; UNSPECIFIED SIDE EFFECT; ANIMAL; GENE EXPRESSION REGULATION; GENE THERAPY; GENETICS; MUSCULAR ATROPHY, SPINAL; MUSCULAR DYSTROPHIES, LIMB-GIRDLE; MUSCULAR DYSTROPHY, DUCHENNE; PATHOPHYSIOLOGY; PROCEDURES; RNA SPLICING; TREATMENT OUTCOME; WALKER-WARBURG SYNDROME;

ANIMALS; EXONS; GENE EXPRESSION REGULATION; GENETIC THERAPY; HUMANS; MUSCULAR ATROPHY, SPINAL; MUSCULAR DYSTROPHIES, LIMB-GIRDLE; MUSCULAR DYSTROPHY, DUCHENNE; OLIGONUCLEOTIDES, ANTISENSE; RNA SPLICING; TREATMENT OUTCOME; WALKER-WARBURG SYNDROME;

EID: 84900835509     PISSN: 14712598     EISSN: 17447682     Source Type: Journal    
DOI: 10.1517/14712598.2014.896335     Document Type: Review

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