Citation
Kiang, Anne, et al. "Fully Deleted Adenovirus Persistently Expressing GAA Accomplishes Long-term Skeletal Muscle Glycogen Correction in Tolerant and Nontolerant GSD-II Mice." Molecular Therapy : the Journal of the American Society of Gene Therapy, vol. 13, no. 1, 2006, pp. 127-34.
Kiang A, Hartman ZC, Liao S, et al. Fully deleted adenovirus persistently expressing GAA accomplishes long-term skeletal muscle glycogen correction in tolerant and nontolerant GSD-II mice. Mol Ther. 2006;13(1):127-34.
Kiang, A., Hartman, Z. C., Liao, S., Xu, F., Serra, D., Palmer, D. J., Ng, P., & Amalfitano, A. (2006). Fully deleted adenovirus persistently expressing GAA accomplishes long-term skeletal muscle glycogen correction in tolerant and nontolerant GSD-II mice. Molecular Therapy : the Journal of the American Society of Gene Therapy, 13(1), 127-34.
Kiang A, et al. Fully Deleted Adenovirus Persistently Expressing GAA Accomplishes Long-term Skeletal Muscle Glycogen Correction in Tolerant and Nontolerant GSD-II Mice. Mol Ther. 2006;13(1):127-34. PubMed PMID: 16169280.
TY - JOUR
T1 - Fully deleted adenovirus persistently expressing GAA accomplishes long-term skeletal muscle glycogen correction in tolerant and nontolerant GSD-II mice.
AU - Kiang,Anne,
AU - Hartman,Zachary C,
AU - Liao,Shaoxi,
AU - Xu,Fang,
AU - Serra,Delila,
AU - Palmer,Donna J,
AU - Ng,Philip,
AU - Amalfitano,Andrea,
Y1 - 2005/10/05/
PY - 2004/11/18/received
PY - 2005/08/01/revised
PY - 2005/08/01/accepted
PY - 2005/9/20/pubmed
PY - 2006/6/13/medline
PY - 2005/9/20/entrez
SP - 127
EP - 34
JF - Molecular therapy : the journal of the American Society of Gene Therapy
JO - Mol Ther
VL - 13
IS - 1
N2 - Glycogen storage disease type II (GSD-II) patients manifest symptoms of muscular dystrophy secondary to abnormal glycogen storage in cardiac and skeletal muscles. For GSD-II, we hypothesized that a fully deleted adenovirus (FDAd) vector expressing hGAA via nonviral regulatory elements (PEPCK promoter/ApoE enhancer) would facilitate long-term efficacy and decrease propensity to generate anti-hGAA antibody responses against hepatically secreted hGAA. Intravenous delivery of FDAdhGAA into GAA-tolerant or nontolerant GAA-KO mice resulted in long-term hepatic secretion of hGAA. Specifically, nontolerant mice achieved complete reversal of cardiac glycogen storage and near-complete skeletal glycogen correction for at least 180 days and tolerant mice for minimally 300 days coupled with the preservation of muscle strength. Anti-hGAA antibody levels in both mouse strains were significantly less relative to those previously generated by CMV-driven hGAA expression in nontolerant GAA-KO mice. However, plasma GAA levels decreased in nontolerant GAA-KO mice despite long-term intrahepatic GAA expression from the persistent vector. This intriguing result is discussed in light of other examples of "tolerance" induction by gene-transfer-based approaches.
SN - 1525-0016
UR - https://www.unboundmedicine.com/medline/citation/16169280/Fully_deleted_adenovirus_persistently_expressing_GAA_accomplishes_long_term_skeletal_muscle_glycogen_correction_in_tolerant_and_nontolerant_GSD_II_mice_
L2 - https://linkinghub.elsevier.com/retrieve/pii/S1525-0016(05)01554-6
DB - PRIME
DP - Unbound Medicine
ER -