Enhanced Factor VIII Heavy Chain for Gene Therapy of Hemophilia A

Hemophilia A gene therapy using recombinant adenovirus-associated virus (AAV) vectors has been hampered by the size of the factor VIII (FVIII) cDNA. Previously, splitting the FVIII coding sequence into a heavy-chain (HC) fragment and a light-chain (LC) fragment for dual recombinant AAV vector delive...

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Bibliografske podrobnosti
Main Authors: Chen, Lingxia, Lu, Hui, Wang, Jinhui, Sarkar, Rita, Yang, Xiao, Wang, Hongli, High, Katherine A, Xiao, Weidong
Format: Artigo
Jezik:Inglês
Izdano: Nature Publishing Group 2009
Teme:
Original Articles
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC2790722/
https://ncbi.nlm.nih.gov/pubmed/19127250
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2008.292
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