Moving towards successful exon-skipping therapy for Duchenne muscular dystrophy

Journal of Human Genetics

Volumn 62, Issue 10, 2017, Pages 871-876

  Nakamura, Akinori ^a,b  

^a National Hospital Organization Matsumoto Medical Center Matsumoto Hospital   (Japan)

^b SHINSHU UNIVERSITY SCHOOL OF MEDICINE   (Japan)

Author keywords

[No Author keywords available]

Indexed keywords

DYSTROPHIN;

DUCHENNE MUSCULAR DYSTROPHY; EXON SKIPPING; GENE THERAPY; GENOTYPE PHENOTYPE CORRELATION; HUMAN; MUSCLE FUNCTION; NONHUMAN; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PHENOTYPE; PRECLINICAL STUDY; PROTEIN EXPRESSION; READING FRAME; REVIEW; ANIMAL; CLINICAL TRIAL (TOPIC); EXON; GENETICS; MUTATION; PROCEDURES; TARGETED GENE REPAIR; TREATMENT OUTCOME;

ANIMALS; CLINICAL TRIALS AS TOPIC; DRUG EVALUATION, PRECLINICAL; DYSTROPHIN; EXONS; GENETIC THERAPY; HUMANS; MUSCULAR DYSTROPHY, DUCHENNE; MUTATION; PHENOTYPE; TARGETED GENE REPAIR; TREATMENT OUTCOME;

EID: 85029841362     PISSN: 14345161     EISSN: 1435232X     Source Type: Journal    
DOI: 10.1038/jhg.2017.57     Document Type: Review

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