Efficient gene transduction in pigs and macaques with the engineered AAV vector AAV.GT5 for hemophilia B gene therapy
Author:
Funder
Japan Agency for Medical Research and Development
Publisher
Elsevier BV
Subject
Genetics,Molecular Biology,Molecular Medicine
Reference37 articles.
1. Hemophilia gene therapy: ushering in a new treatment paradigm?;George;Hematology. Am. Soc. Hematol. Educ. Program,2021
2. Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B;Nathwani;N. Engl. J. Med.,2011
3. Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant;George;N. Engl. J. Med.,2017
4. Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B;Miesbach;Blood,2018
5. Phase 1–2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B;Chowdary;N. Engl. J. Med.,2022
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