Future of rAAV Gene Therapy: Platform for RNAi, Gene Editing, and Beyond
Author:
Affiliation:
1. Departments of Pediatrics and Genetics, Stanford University, Stanford, California.
Publisher
Mary Ann Liebert Inc
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
http://www.liebertpub.com/doi/pdf/10.1089/hum.2016.171
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1. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors
2. Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
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5. Vision 1 Year after Gene Therapy for Leber's Congenital Amaurosis
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