Development of a Clinical Candidate AAV3 Vector for Gene Therapy of Hemophilia B

Although recombinant adeno-associated virus serotype 8 (AAV8) and serotype 5 (AAV5) vectors have shown efficacy in Phase 1 clinical trials for gene therapy of hemophilia B, it has become increasingly clear that these serotypes are not optimal for transducing primary human hepatocytes. We have previo...

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Bibliografiske detaljer
Udgivet i:Hum Gene Ther
Main Authors: Brown, Harrison C., Doering, Christopher B., Herzog, Roland W., Ling, Chen, Markusic, David M., Spencer, H. Trent, Srivastava, Alok, Srivastava, Arun
Format: Artigo
Sprog:Inglês
Udgivet: Mary Ann Liebert, Inc., publishers 2020
Fag:
Research Articles
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7585622/
https://ncbi.nlm.nih.gov/pubmed/32657150
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2020.099
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