Exon skipping: a first in class strategy for Duchenne muscular dystrophy

Expert Opinion on Biological Therapy

Volumn 17, Issue 2, 2017, Pages 225-236

  Niks, Erik H ^a   Aartsma Rus, Annemieke ^a  

^a LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

Author keywords

antisense oligonucleotide; DMD; dystrophin; splicing; therapy

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DYSTROPHIN;

ALTERNATIVE RNA SPLICING; CRISPR CAS SYSTEM; DRUG CONJUGATION; DRUG FORMULATION; DUCHENNE MUSCULAR DYSTROPHY; EXON SKIPPING; GENE DELETION; GENE DUPLICATION; GENE MUTATION; HUMAN; INTRON; MULTIEXON SKIPPING; NONHUMAN; PHASE 2 CLINICAL TRIAL (TOPIC); RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; ANIMAL; CLINICAL TRIAL (TOPIC); EXON; GENE THERAPY; GENETICS; MUSCULAR DYSTROPHY, DUCHENNE; PROCEDURES; RNA SPLICING; TRENDS;

ANIMALS; CLINICAL TRIALS AS TOPIC; DYSTROPHIN; EXONS; GENETIC THERAPY; HUMANS; MUSCULAR DYSTROPHY, DUCHENNE; OLIGONUCLEOTIDES, ANTISENSE; RNA SPLICING;

EID: 85009977190     PISSN: 14712598     EISSN: 17447682     Source Type: Journal    
DOI: 10.1080/14712598.2017.1271872     Document Type: Review

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