Ontology highlight
ABSTRACT:
SUBMITTER: Mueller C
PROVIDER: S-EPMC3859421 | biostudies-literature | 2013 Dec
REPOSITORIES: biostudies-literature
Mueller Christian C Chulay Jeffrey D JD Trapnell Bruce C BC Humphries Margaret M Carey Brenna B Sandhaus Robert A RA McElvaney Noel G NG Messina Louis L Tang Qiushi Q Rouhani Farshid N FN Campbell-Thompson Martha M Fu Ann Dongtao AD Yachnis Anthony A Knop David R DR Ye Guo-Jie GJ Brantly Mark M Calcedo Roberto R Somanathan Suryanarayan S Richman Lee P LP Vonderheide Robert H RH Hulme Maigan A MA Brusko Todd M TM Wilson James M JM Flotte Terence R TR
The Journal of clinical investigation 20131115 12
Recombinant adeno-associated virus (rAAV) vectors have shown promise for the treatment of several diseases; however, immune-mediated elimination of transduced cells has been suggested to limit and account for a loss of efficacy. To determine whether rAAV vector expression can persist long term, we administered rAAV vectors expressing normal, M-type α-1 antitrypsin (M-AAT) to AAT-deficient subjects at various doses by multiple i.m. injections. M-specific AAT expression was observed in all subject ...[more]