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

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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Bibliographic Details
Main Authors: Sandeep R.P. Kumar, Jun Xie, Shilang Hu, Jihye Ko, Qifeng Huang, Harrison C. Brown, Alok Srivastava, David M. Markusic, Christopher B. Doering, H. Trent Spencer, Arun Srivastava, Guangping Gao, Roland W. Herzog
Format: Article
Language:English
Published: Elsevier 2021-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
adeno-associated virus
AAV
hemophilia
factor IX
liver
non-human primate
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121001273

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http://www.sciencedirect.com/science/article/pii/S2329050121001273

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