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Molecular Therapy. Methods & Clinical Development|October 8, 2015
AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypesDaniel J Hui, Shyrie C Edmonson, Gregory M Podsakoff, et al.
Human Gene Therapy|August 7, 2002
Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapyRoland W Herzog, Paul A Fields, Valder R Arruda, et al.
Journal of Thrombosis and Haemostasis : JTH|February 26, 2021
Factor IX assay discrepancies in the setting of liver gene therapy using a hyperfunctional variant factor IX-PaduaMary M Robinson, Lindsey A George, Marcus E Carr, et al.
Blood|December 25, 2015
Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVIIOscar A Marcos-Contreras, Shannon M Smith, Dwight A Bellinger, et al.
Ophthalmology|April 2, 2021
Durability of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease: Phase 3 Results at 3 and 4 YearsAlbert M Maguire, Stephen Russell, Daniel C Chung, et al.
Blood|November 26, 2010
Assessing the potential for AAV vector genotoxicity in a murine modelHojun Li, Nirav Malani, Shari R Hamilton, et al.
Human Gene Therapy|May 6, 2011
Cardiac gene transfer of short hairpin RNA directed against phospholamban effectively knocks down gene expression but causes cellular toxicity in caninesLawrence T Bish, Meg M Sleeper, Caryn Reynolds, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|July 11, 2006
Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia BHaiyan Jiang, Glenn F Pierce, Margareth C Ozelo, et al.
Blood|April 4, 2002
Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapyJane D Mount, Roland W Herzog, D Michael Tillson, et al.
AIDS (London, England)|December 13, 2002
Platelet- and megakaryocyte-derived microparticles transfer CXCR4 receptor to CXCR4-null cells and make them susceptible to infection by X4-HIVTomasz Rozmyslowicz, Marcin Majka, Jacek Kijowski, et al.
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