Progress and prospects of gene therapy clinical trials for the muscular dystrophies

Human Molecular Genetics

Volumn 25, Issue R1, 2016, Pages R9-R17

  Bengtsson, Niclas E ^a   Seto, Jane T ^a,c   Hall, John K ^a   Chamberlain, Jeffrey S ^a,b   Odom, Guy L ^a  

^a UNIVERSITY OF WASHINGTON   (United States)

^b UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

^c ROYAL CHILDREN'S HOSPITAL   (Australia)

Author keywords

[No Author keywords available]

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; ALPHA SARCOGLYCAN; DYSFERLIN; DYSTROPHIN; FUKUTIN RELATED PROTEIN;

CLINICAL TRIAL (TOPIC); DUCHENNE MUSCULAR DYSTROPHY; DYSFERLINOPATHY; GENE EDITING; GENE THERAPY; HUMAN; IMMUNE RESPONSE; LIMB GIRDLE MUSCULAR DYSTROPHY; MUSCULAR DYSTROPHY; NONHUMAN; PRECLINICAL STUDY; PRIORITY JOURNAL; REVIEW; SARCOGLYCANOPATHY; VIRUS CAPSID; MUSCULAR DYSTROPHIES;

CLINICAL TRIALS AS TOPIC; GENETIC THERAPY; HUMANS; MUSCULAR DYSTROPHIES;

EID: 85011728863     PISSN: 09646906     EISSN: 14602083     Source Type: Journal    
DOI: 10.1093/hmg/ddv420     Document Type: Review

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