AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

By S. Rangarajan, Liron Walsh, W. Lester, D. Perry, B. Madan, M. Laffan, Hua Yu, Christian Vettermann, G. Pierce, W. Wong and 1 more. New England Journal of Medicine, 2017

602 citations recorded by the source corpus.

Subfields: Genetics, Hematology

View Paper: https://www.semanticscholar.org/paper/42d752316e35d176fab9cd6f36e29a2910a9eb2b

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In Genetics

1. K. High
2. J. Keith Joung
3. Guangping Gao
4. Wei Zhan
5. Thomas L. Gallagher
6. Jiang-Hui Wang
7. K. Ozawa
8. C. Dunbar

Citations in the corpus

• Gene therapy comes of age (2018, positive): “Results from the first positive early-phase clinical trial for hemophilia A were recently reported, demonstrating increases in factor VIII activity into or even above the normal range in six of seven patients, accompanied by decreased bleeding (89).”
• Adeno-associated virus as a delivery vector for gene therapy of human diseases (2024, background): “Although vector properties between the trials by Savita et al. 312 and George et al. 314 might explain the difference in transgene expression and maintenance, it was hypothesized that high expression levels might trigger an unfolded protein response with subsequent FVIII loss.”
• Current Clinical Applications of In Vivo Gene Therapy with AAVs (2020, background): “, 2020, WFH Virtual Summit, conference).(145,149,150,153) Part of this variability may be explained, in part, by a hypothesized cellular immune response to capsid peptides presented by transduced hepatocytes that can result in reduced or loss of transgene expression, thereby limiting efficacy.”