Complete Correction of Hemophilia A with Adeno-Associated Viral Vectors Containing a Full-Size Expression Cassette

Hemophilia A is caused by a deficiency in the factor VIII (FVIII) gene. Constrained by limited packaging capacity, even the 4.3-kb B domain-deleted FVIII remained a challenge for delivery by a single adeno-associated viral (AAV) vector. Studies have shown that up to a 6.6-kb vector sequence may be p...

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Bibliografiske detaljer
Main Authors: Lu, Hui, Chen, Lingxia, Wang, Jinhui, Huack, Bernd, Sarkar, Rita, Zhou, Shangzhen, Xu, Ray, Ding, Qiulan, Wang, Xuefeng, Wang, Hongli, Xiao, Weidong
Format: Artigo
Sprog:Inglês
Udgivet: Mary Ann Liebert, Inc. 2008
Fag:
Brief Report
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2596974/
https://ncbi.nlm.nih.gov/pubmed/18500941
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2007.0182
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