AAV vectors for long-term gene therapy of hemophilia B: Are we there yet?
Author:
Funder
Kitzman Foundation
U.S. Public Health Service
National Institutes of Health
Children's Miracle Network Hospitals
Publisher
Elsevier BV
Reference23 articles.
1. 2022. https://www.fda.gov/vaccines-blood-biologics/vaccines/hemgenix.
2. 2024. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/beqvez
3. Correlation of antigen expression with epigenetic modifications after rAAV delivery of a human factor IX variant in mice and rhesus macaques
4. Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response;Manno;Nat. Med.,2006
5. Long-term safety and efficacy of factor IX gene therapy in hemophilia B;Nathwani;N. Engl. J. Med.,2014
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