Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning

Human Gene Therapy Clinical Development

Volumn 26, Issue 3, 2015, Pages 185-193

  Corti, Manuela ^a   Cleaver, Brian ^a   Clément, Nathalie ^a   Conlon, Thomas J ^a   Faris, Kaitlyn J ^a   Wang, Gensheng ^b   Benson, Janet ^b   Tarantal, Alice F ^c,d   Fuller, Davis ^e   Herzog, Roland W ^a   Byrne, Barry J ^a  

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

^b LOVELACE RESPIRATORY RESEARCH INSTITUTE   (United States)

^c UNIVERSITY OF CALIFORNIA   (United States)

^d UNIVERSITY OF CALIFORNIA   (United States)

^e UNIVERSITY OF FLORIDA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; DESMIN; GLUCAN 1,4 ALPHA GLUCOSIDASE; NEUTRALIZING ANTIBODY; PLACEBO; RAPAMYCIN; RITUXIMAB; IMMUNOLOGIC FACTOR; VIRUS ANTIBODY;

ADENO ASSOCIATED VIRUS 9; ADULT; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIBODY PRODUCTION; ARTICLE; CLINICAL ARTICLE; CONTROLLED STUDY; DOUBLE BLIND PROCEDURE; DRUG DISTRIBUTION; DRUG SAFETY; FEMALE; GLYCOGEN STORAGE DISEASE TYPE 2; HEART MUSCLE; HUMAN; IMMUNOGENICITY; IMMUNOMODULATION; MALE; MOUSE; NONHUMAN; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; REPEATED DRUG DOSE; RHESUS MONKEY; SKELETAL MUSCLE; TIBIALIS ANTERIOR MUSCLE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS RECOMBINANT; ADMINISTRATION AND DOSAGE; ADVERSE EFFECTS; ANIMAL; BLOOD; CLASSIFICATION; CLINICAL PROTOCOL; DEPENDOPARVOVIRUS; DISEASE MODEL; DRUG EFFECTS; GENE EXPRESSION; GENE VECTOR; GENETICS; GLYCOGEN STORAGE DISEASE TYPE II; IMMUNOLOGY; INTRAMUSCULAR DRUG ADMINISTRATION; KNOCKOUT MOUSE; METABOLISM; PHARMACOKINETICS; RETREATMENT; TISSUE DISTRIBUTION;

ADULT; ANIMALS; ANTIBODIES, VIRAL; CLINICAL PROTOCOLS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; FEMALE; GENE EXPRESSION; GENETIC VECTORS; GLUCAN 1,4-ALPHA-GLUCOSIDASE; GLYCOGEN STORAGE DISEASE TYPE II; HUMANS; IMMUNOLOGIC FACTORS; IMMUNOMODULATION; INJECTIONS, INTRAMUSCULAR; MACACA MULATTA; MALE; MICE; MICE, KNOCKOUT; RETREATMENT; RITUXIMAB; TISSUE DISTRIBUTION;

EID: 85006208454     PISSN: 23248637     EISSN: 23248645     Source Type: Journal    
DOI: 10.1089/humc.2015.068     Document Type: Article

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