Phase I clinical trial utilizing gene therapy for limb girdle muscular dystrophy: α-, β-, γ-, or Δ-sarcoglycan gene delivered with intramuscular instillations of adeno-associated vectors

Human Gene Therapy

Volumn 11, Issue 5, 2000, Pages 777-790

  Stedman, Hansell ^a   Wilson, James M ^b   Finke, Richard ^c   Kleckner, Anna Lisa ^d   Mendell, Jerry ^e  

^a HUP   (United States)

^b Institute for Human Gene Therapy   (United States)

^c CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^d UNIVERSITY OF PENNSYLVANIA   (United States)

^e OHIO STATE UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

PLACEBO; SARCOGLYCAN; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; CLINICAL PROTOCOL; CLINICAL TRIAL; DOUBLE BLIND PROCEDURE; GENE THERAPY; HUMAN; LIMB GIRDLE MUSCULAR DYSTROPHY; MAXIMUM PERMISSIBLE DOSE; METHODOLOGY; NOTE; PATIENT SELECTION; PHASE 1 CLINICAL TRIAL;

ADENOVIRIDAE; ANIMALS; CLINICAL PROTOCOLS; CLINICAL TRIALS, PHASE I; CYTOSKELETAL PROTEINS; DYSTROGLYCANS; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; INJECTIONS, INTRAMUSCULAR; MEMBRANE GLYCOPROTEINS; MICE; MICE, KNOCKOUT; MUSCULAR DYSTROPHIES; SARCOGLYCANS;

ADENO-ASSOCIATED VIRUS;

EID: 0034689207     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/10430340050015671     Document Type: Note

References (0)