Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette

Hepatic gene transfer with adeno-associated viral (AAV) vectors shows much promise for the treatment of the X-linked bleeding disorder hemophilia B in multiple clinical trials. In an effort to further innovate this approach and to introduce alternative vector designs with potentially superior featur...

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Detalles Bibliográficos
Autores principales: Kumar, Sandeep R.P., Xie, Jun, Hu, Shilang, Ko, Jihye, Huang, Qifeng, Brown, Harrison C., Srivastava, Alok, Markusic, David M., Doering, Christopher B., Spencer, H. Trent, Srivastava, Arun, Gao, Guangping, Herzog, Roland W.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8476648/
https://www.ncbi.nlm.nih.gov/pubmed/34631930
http://dx.doi.org/10.1016/j.omtm.2021.08.001

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