Limb-girdle muscular dystrophy type 2D gene therapy restores α-sarcoglycan and associated proteins

Annals of Neurology

Volumn 66, Issue 3, 2009, Pages 290-297

  Mendell, Jerry R ^a,b   Rodino Klapac, Louise R ^a,b   Rosales Quintero, Xiomara ^a,b   Kota, Janaiah ^a,b   Coley, Brian D ^a,b   Galloway, Gloria ^a,b   Craenen, Josepha M ^a,b   Lewis, Sarah ^b   Malik, Vinod ^b   Shilling, Christopher ^a,b   Byrne, Barry J ^c,d   Conlon, Thomas ^c,d   Campbell, Katherine J ^b   Bremer, William G ^b   Viollet, Laurence ^b   Walker, Christopher M ^a,b   Sahenk, Zarife ^a,b   Reed Clark, K ^a,b  

^a OHIO STATE UNIVERSITY   (United States)

^b NATIONWIDE CHILDREN S HOSPITAL   (United States)

^c UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

^d UNIVERSITY OF FLORIDA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA SARCOGLYCAN; CD4 ANTIGEN; CD8 ANTIGEN; GAMMA INTERFERON; GENE PRODUCT; MAJOR HISTOCOMPATIBILITY ANTIGEN CLASS 1; METHYLPREDNISOLONE; MUSCLE SPECIFIC CREATINE KINASE HUMAN ALPHA SARCOGLYCAN RECOMBINANT ADENO ASSOCIATED VIRUS 1 VECTOR; NEUTRALIZING ANTIBODY; UNCLASSIFIED DRUG;

ADENO ASSOCIATED VIRUS; ADENO ASSOCIATED VIRUS SEROTYPE 1; ADOLESCENT; ANTIBODY TITER; APOPTOSIS; ARTICLE; CLINICAL ARTICLE; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DISEASE SEVERITY; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; DRUG MEGADOSE; DRUG SAFETY; ENZYME LINKED IMMUNOSPOT ASSAY; GENE; GENE EXPRESSION; HEPATOMEGALY; HUMAN; HUMAN CELL; HUMAN TISSUE; IMMUNE RESPONSE; IMMUNOTHERAPY; INFLAMMATION; INJECTION SITE ERYTHEMA; INJECTION SITE PAIN; INJECTION SITE RASH; INJECTION SITE SWELLING; LIMB GIRDLE MUSCULAR DYSTROPHY; LIMB GIRDLE MUSCULAR DYSTROPHY TYPE 2D; LOW DRUG DOSE; LYMPHADENOPATHY; NONHUMAN; PERIPHERAL BLOOD MONONUCLEAR CELL; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; SGCA GENE; SPLENOMEGALY; T LYMPHOCYTE; TREATMENT OUTCOME; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS CAPSID;

EID: 70350067897     PISSN: 03645134     EISSN: 15318249     Source Type: Journal    
DOI: 10.1002/ana.21732     Document Type: Article

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