Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease

Human Gene Therapy

Volumn 27, Issue 1, 2016, Pages 43-59

  Doerfler, Phillip A ^a   Todd, Adrian G ^a   Clément, Nathalie ^a   Falk, Darin J ^a   Nayak, Sushrusha ^b   Herzog, Roland W ^a   Byrne, Barry J ^a  

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

^b KAROLINSKA INSTITUTE   (Sweden)

Author keywords

[No Author keywords available]

Indexed keywords

DESMIN; GLUCAN 1,4 ALPHA GLUCOSIDASE;

ADENO ASSOCIATED VIRUS 9; ADOPTIVE TRANSFER; ALLERGIC REACTION; ANAPHYLAXIS; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIGEN SPECIFICITY; ARTICLE; CELLULAR IMMUNITY; CODON; CONTROLLED STUDY; ENZYME ACTIVITY; ENZYME REPLACEMENT; FEMALE; GLYCOGEN STORAGE DISEASE TYPE 2; HEART MUSCLE; HUMORAL IMMUNITY; IMMUNE RESPONSE; IMMUNITY; IMMUNOLOGICAL TOLERANCE; LIVER; MALE; MOUSE; NONHUMAN; PERIPHERAL NERVE; PHENOTYPE; PROMOTER REGION; REGULATORY T LYMPHOCYTE; RISK ASSESSMENT; SKELETAL MUSCLE; SPINAL CORD; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; ANIMAL; DEFICIENCY; DEPENDOPARVOVIRUS; GENE THERAPY; GENETICS; GLYCOGEN STORAGE DISEASE TYPE II; HUMAN; IMMUNOLOGY; KNOCKOUT MOUSE; METABOLISM;

ANIMALS; DEPENDOVIRUS; GENETIC THERAPY; GLUCAN 1,4-ALPHA-GLUCOSIDASE; GLYCOGEN STORAGE DISEASE TYPE II; HUMANS; IMMUNE TOLERANCE; MICE; MICE, KNOCKOUT; PROMOTER REGIONS, GENETIC; T-LYMPHOCYTES, REGULATORY;

EID: 84955262547     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2015.103     Document Type: Article

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