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Molecular Therapy : the Journal of the American Society of Gene Therapy|March 29, 2025
Gene therapy for hemophilia - From basic science to first approvals of "one-and-done" therapiesRoland W Herzog, Radoslaw Kaczmarek, Katherine A HighCurrent Gene Therapy|August 18, 2010
Manufacturing and regulatory strategies for clinical AAV2-hRPE65J Fraser Wright, Jennifer Wellman, Katherine A HighHematology. American Society of Hematology. Education Program|November 10, 2001
Congenital Hemorrhagic Disorders: New Insights into the Pathophysiology and Treatment of HemophiliaUlla Hedner, David Ginsburg, Jeanne M. Lusher, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|May 9, 2012
Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia BFederico Mingozzi, Yifeng Chen, Samuel L Murphy, et al.The Journal of Clinical Investigation|May 14, 2009
Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectorsGary C Pien, Etiena Basner-Tschakarjan, Daniel J Hui, et al.Journal of Virology|July 20, 2007
TR1.3 viral pathogenesis and syncytium formation are linked to Env-Gag cooperationSamuel L Murphy, Glen N GaultonThe Biochemical Journal|April 5, 2002
A frequent human coagulation Factor VII mutation (A294V, c152) in loop 140s affects the interaction with activators, tissue factor and substratesRaffaella Toso, Mirko Pinotti, Katherine A High, et al.American Journal of Hematology|November 29, 2011
Clinical trial opportunities in hemostasis and thrombosis: NHBLI State-of-the-Science symposiumDouglas B Cines, Morris A Blajchman, Katherine A High, et al.Nature Medicine|March 21, 2007
CD8(+) T-cell responses to adeno-associated virus capsid in humansFederico Mingozzi, Marcela V Maus, Daniel J Hui, et al.Journal of Virological Methods|February 8, 2011
PEG-modulated column chromatography for purification of recombinant adeno-associated virus serotype 9Jingmin Zhou, Xiao Yang, J Fraser Wright, et al.Pageof 15