Citation
Sun, Baodong, et al. "Efficacy of an Adeno-associated Virus 8-pseudotyped Vector in Glycogen Storage Disease Type II." Molecular Therapy : the Journal of the American Society of Gene Therapy, vol. 11, no. 1, 2005, pp. 57-65.
Sun B, Zhang H, Franco LM, et al. Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II. Mol Ther. 2005;11(1):57-65.
Sun, B., Zhang, H., Franco, L. M., Young, S. P., Schneider, A., Bird, A., Amalfitano, A., Chen, Y. T., & Koeberl, D. D. (2005). Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II. Molecular Therapy : the Journal of the American Society of Gene Therapy, 11(1), 57-65.
Sun B, et al. Efficacy of an Adeno-associated Virus 8-pseudotyped Vector in Glycogen Storage Disease Type II. Mol Ther. 2005;11(1):57-65. PubMed PMID: 15585406.
TY - JOUR
T1 - Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II.
AU - Sun,Baodong,
AU - Zhang,Haoyue,
AU - Franco,Luis M,
AU - Young,Sarah P,
AU - Schneider,Ayn,
AU - Bird,Andrew,
AU - Amalfitano,Andrea,
AU - Chen,Y-T,
AU - Koeberl,Dwight D,
PY - 2004/06/14/received
PY - 2004/10/01/revised
PY - 2004/10/05/accepted
PY - 2004/12/9/pubmed
PY - 2005/5/5/medline
PY - 2004/12/9/entrez
SP - 57
EP - 65
JF - Molecular therapy : the journal of the American Society of Gene Therapy
JO - Mol Ther
VL - 11
IS - 1
N2 - Glycogen storage disease type II (GSD-II; Pompe disease) causes death in infancy from cardiorespiratory failure. The underlying deficiency of acid alpha-glucosidase (GAA; acid maltase) can be corrected by liver-targeted gene therapy in GSD-II, if secretion of GAA is accompanied by receptor-mediated uptake in cardiac and skeletal muscle. An adeno-associated virus (AAV) vector encoding human (h) GAA was pseudotyped as AAV8 (AAV2/8) and injected intravenously into immunodeficient GSD-II mice. High levels of hGAA were maintained in plasma for 24 weeks following AAV2/8 vector administration. A marked increase in vector copy number in the liver was demonstrated for the AAV2/8 vector compared to the analogous AAV2/2 vector. GAA deficiency in the heart and skeletal muscle was corrected with the AAV2/8 vector in male GSD-II mice, consistent with receptor-mediated uptake of hGAA. Male GSD-II mice demonstrated complete correction of glycogen storage in heart and diaphragm with the AAV2/8 vector, while female GSD-II mice had correction only in the heart. A biomarker for GSD-II was reduced in both sexes following AAV2/8 vector administration. Therefore, GAA production with an AAV2/8 vector in a depot organ, the liver, generated evidence for efficacious gene therapy in a mouse model for GSD-II.
SN - 1525-0016
UR - https://www.unboundmedicine.com/medline/citation/15585406/Efficacy_of_an_adeno_associated_virus_8_pseudotyped_vector_in_glycogen_storage_disease_type_II_
L2 - https://linkinghub.elsevier.com/retrieve/pii/S1525-0016(04)01485-6
DB - PRIME
DP - Unbound Medicine
ER -