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Molecular Therapy : the Journal of the American Society of Gene Therapy|January 27, 2020
Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and HumansGwladys Gernoux, Alisha M Gruntman, Meghan Blackwood, et al.Journal of Cellular Physiology|June 20, 2007
Gene therapy: the first two decades and the current state-of-the-artTerence R FlottePediatric Research|November 25, 2005
Adeno-associated virus-based gene therapy for inherited disordersTerence R FlotteCurrent Gene Therapy|June 25, 2005
Recent developments in recombinant AAV-mediated gene therapy for lung diseasesTerence R FlotteMolecular Therapy. Methods & Clinical Development|May 20, 2022
Liver-directed SERPINA1 gene therapy attenuates progression of spontaneous and tobacco smoke-induced emphysema in α1-antitrypsin null miceMarina Zieger, Florie Borel, Cynthia Greer, et al.The Journal of Gene Medicine|November 21, 2007
Partial correction of the CFTR-dependent ABPA mouse model with recombinant adeno-associated virus gene transfer of truncated CFTR geneChristian Mueller, Daniel Torrez, Sofia Braag, et al.Current Protocols in Microbiology|February 15, 2013
Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virusAlisha M Gruntman, Lawrence T Bish, Christian Mueller, et al.International Journal of Molecular Sciences|January 23, 2024
Delivery of Adeno-Associated Virus Vectors to the Central Nervous System for Correction of Single Gene DisordersRrita Daci, Terence R FlotteAmerican 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.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.Pageof 90