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Molecular Therapy. Methods & Clinical Development|October 3, 2022
Selection of rAAV vectors that cross the human blood-brain barrier and target the central nervous system using a transwell modelRen Song, Katja Pekrun, Themasap A Khan, et al.Science Translational Medicine|June 10, 2016
A universal system to select gene-modified hepatocytes in vivoSean Nygaard, Adi Barzel, Annelise Haft, et al.American Journal of Physiology. Endocrinology and Metabolism|June 10, 2010
FATP2 is a hepatic fatty acid transporter and peroxisomal very long-chain acyl-CoA synthetaseAlaric Falcon, Holger Doege, Amy Fluitt, et al.Molecular Therapy. Nucleic Acids|February 23, 2023
Aptamer-programmable adeno-associated viral vectors as a novel platform for cell-specific gene transferFrancesco Puzzo, Chuanling Zhang, Bethany Powell Gray, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|December 14, 2006
Somatic integration from an adenoviral hybrid vector into a hot spot in mouse liver results in persistent transgene expression levels in vivoAnja Ehrhardt, Stephen R Yant, Jeffery C Giering, et al.Nature Structural & Molecular Biology|August 4, 2014
Regulation of microRNA-mediated gene silencing by microRNA precursorsBiswajoy Roy-Chaudhuri, Paul N Valdmanis, Yue Zhang, et al.Proceedings of the National Academy of Sciences of the United States of America|May 18, 2011
Thermodynamic stability of small hairpin RNAs highly influences the loading process of different mammalian ArgonautesShuo Gu, Lan Jin, Feijie Zhang, et al.Cell|November 13, 2012
The loop position of shRNAs and pre-miRNAs is critical for the accuracy of dicer processing in vivoShuo Gu, Lan Jin, Yue Zhang, et al.Blood|June 14, 2003
A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopeniaAnja Ehrhardt, Hui Xu, Aaron M Dillow, et al.Nature Biotechnology|October 16, 2002
Site-specific genomic integration produces therapeutic Factor IX levels in miceEric C Olivares, Roger P Hollis, Thomas W Chalberg, et al.Pageof 19