Citation
Franco, Luis M., et al. "Evasion of Immune Responses to Introduced Human Acid Alpha-glucosidase By Liver-restricted Expression in Glycogen Storage Disease Type II." Molecular Therapy : the Journal of the American Society of Gene Therapy, vol. 12, no. 5, 2005, pp. 876-84.
Franco LM, Sun B, Yang X, et al. Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II. Mol Ther. 2005;12(5):876-84.
Franco, L. M., Sun, B., Yang, X., Bird, A., Zhang, H., Schneider, A., Brown, T., Young, S. P., Clay, T. M., Amalfitano, A., Chen, Y. T., & Koeberl, D. D. (2005). Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II. Molecular Therapy : the Journal of the American Society of Gene Therapy, 12(5), 876-84.
Franco LM, et al. Evasion of Immune Responses to Introduced Human Acid Alpha-glucosidase By Liver-restricted Expression in Glycogen Storage Disease Type II. Mol Ther. 2005;12(5):876-84. PubMed PMID: 16005263.
TY - JOUR
T1 - Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II.
AU - Franco,Luis M,
AU - Sun,Baodong,
AU - Yang,Xiaoyi,
AU - Bird,Andrew,
AU - Zhang,Haoyue,
AU - Schneider,Ayn,
AU - Brown,Talmage,
AU - Young,Sarah P,
AU - Clay,Timothy M,
AU - Amalfitano,Andrea,
AU - Chen,Y T,
AU - Koeberl,Dwight D,
Y1 - 2005/07/06/
PY - 2004/10/01/received
PY - 2005/04/28/revised
PY - 2005/04/28/accepted
PY - 2005/7/12/pubmed
PY - 2006/1/7/medline
PY - 2005/7/12/entrez
SP - 876
EP - 84
JF - Molecular therapy : the journal of the American Society of Gene Therapy
JO - Mol. Ther.
VL - 12
IS - 5
N2 - Glycogen storage disease type II (GSD-II; Pompe disease) is caused by a deficiency of acid alpha-glucosidase (GAA; acid maltase) and manifests as muscle weakness, hypertrophic cardiomyopathy, and respiratory failure. Adeno-associated virus vectors containing either a liver-specific promoter (LSP) (AAV-LSPhGAApA) or a hybrid CB promoter (AAV-CBhGAApA) to drive human GAA expression were pseudotyped as AAV8 and administered to immunocompetent GAA-knockout mice. Secreted hGAA was detectable in plasma between 1 day and 12 weeks postadministration with AAV-LSPhGAApA and only from 1 to 8 days postadministration for AAV-CBGAApA. No anti-GAA antibodies were detected in response to AAV-LSPhGAApA (<1:200), whereas AAV-CBhGAApA provoked an escalating antibody response starting 2 weeks postadministration. The LSP drove approximately 60-fold higher GAA expression than the CB promoter in the liver by 12 weeks following vector administration. Furthermore, the detected cellular immunity was provoked by AAV-CBhGAApA, as detected by ELISpot and CD4+/CD8+ lymphocyte immunodetection. GAA activity was increased to higher than normal and glycogen content was reduced to essentially normal levels in the heart and skeletal muscle following administration of AAV-LSPhGAApA. Therefore, liver-restricted GAA expression with an AAV vector evaded immunity and enhanced efficacy in GSD-II mice.
SN - 1525-0016
UR - https://www.unboundmedicine.com/medline/citation/16005263/Evasion_of_immune_responses_to_introduced_human_acid_alpha_glucosidase_by_liver_restricted_expression_in_glycogen_storage_disease_type_II_
L2 - https://linkinghub.elsevier.com/retrieve/pii/S1525-0016(05)00231-5
DB - PRIME
DP - Unbound Medicine
ER -
