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Molecular Therapy : the Journal of the American Society of Gene Therapy|January 19, 2012
Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profilesChristian Mueller, Qiushi Tang, Alisha Gruntman, et al.
American Journal of Respiratory Cell and Molecular Biology|August 10, 2010
N-glycosylation augmentation of the cystic fibrosis epithelium improves Pseudomonas aeruginosa clearanceAshley T Martino, Christian Mueller, Sofia Braag, et al.
Human Gene Therapy|December 10, 2015
Recombinant Adeno-Associated Virus Vector Genomes Take the Form of Long-Lived, Transcriptionally Competent Episomes in Human MuscleBruce C Schnepp, Jeffrey D Chulay, Guo-Jie Ye, et al.
The Journal of Gene Medicine|August 23, 2008
Recombinant adeno-associated virus-mediated gene delivery of long chain acyl coenzyme A dehydrogenase (LCAD) into LCAD-deficient miceStuart G Beattie, Eric Goetzman, Qiuishi Tang, et al.
Proceedings of the National Academy of Sciences of the United States of America|February 10, 2004
DNA-dependent PK inhibits adeno-associated virus DNA integrationSihong Song, Yuanqing Lu, Young-Kook Choi, et al.
Human Gene Therapy|July 1, 2008
Apparently nonspecific enzyme elevations after portal vein delivery of recombinant adeno-associated virus serotype 2 vector in hepatitis C virus-infected chimpanzeesTerence R Flotte, Jason Goetzmann, James Caridi, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|July 4, 2002
Improved method of recombinant AAV2 delivery for systemic targeted gene therapyCathryn Mah, Thomas J Fraites, Irene Zolotukhin, et al.
Human Gene Therapy|February 18, 2004
Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adultsTerence R Flotte, Mark L Brantly, L Terry Spencer, et al.
American Journal of Respiratory Cell and Molecular Biology|January 27, 2009
Defective acid sphingomyelinase pathway with Pseudomonas aeruginosa infection in cystic fibrosisHong Yu, Youssef H Zeidan, Bill X Wu, et al.
Human Gene Therapy|September 9, 2004
Functional characterization of a recombinant adeno-associated virus 5-pseudotyped cystic fibrosis transmembrane conductance regulator vectorJeffrey Sirninger, Christian Muller, Sofia Braag, et al.
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