Translational development of splice-modifying antisense oligomers

Expert Opinion on Biological Therapy

Volumn 17, Issue 1, 2017, Pages 15-30

  Fletcher, S ^a,b,c   Bellgard, M ^b,c   Price, L ^a,b,c   Akkari, A ^b,c,d   Wilton, S ^a,b,c  

^a UNIVERSITY OF WESTERN AUSTRALIA   (Australia)

^b Western Australian Biomedical Research Institute   (Australia)

^c MURDOCH UNIVERSITY   (Australia)

^d Shiraz Pharmaceuticals   (United States)

Author keywords

adaptive clinical trial; alternative splicing; antisense oligomer; Duchenne muscular dystrophy; dystrophin; exon skipping; Rare diseases; spinal muscular atrophy

Indexed keywords

OLIGOMER; SPLICE MODIFYING ANTISENSE OLIGOMER; UNCLASSIFIED DRUG; ANTISENSE OLIGONUCLEOTIDE; DYSTROPHIN;

DISEASE COURSE; DUCHENNE MUSCULAR DYSTROPHY; EXON; EXON SKIPPING; FATALITY; HUMAN; NONHUMAN; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PHENOTYPE; REVIEW; SPINAL MUSCULAR ATROPHY; VALIDATION STUDY; ANIMAL; BIOLOGICAL THERAPY; DRUG EFFECTS; GENE EXPRESSION; GENE EXPRESSION REGULATION; GENE THERAPY; GENETICS; MUSCULAR ATROPHY, SPINAL; MUSCULAR DYSTROPHY, DUCHENNE; PROCEDURES; RNA SPLICING; TRANSLATIONAL RESEARCH; TRENDS;

ANIMALS; BIOLOGICAL THERAPY; DYSTROPHIN; EXONS; GENE EXPRESSION; GENE EXPRESSION REGULATION; GENETIC THERAPY; HUMANS; MUSCULAR ATROPHY, SPINAL; MUSCULAR DYSTROPHY, DUCHENNE; OLIGONUCLEOTIDES, ANTISENSE; RNA SPLICING; TRANSLATIONAL MEDICAL RESEARCH;

EID: 85002910217     PISSN: 14712598     EISSN: 17447682     Source Type: Journal    
DOI: 10.1080/14712598.2017.1250880     Document Type: Review

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