Advances of adeno-associated virus applied in gene therapy to hemophilia from bench work to the clinical use

Abstract. Hemophilia A and B are diseases caused by a single gene deficiency and are thus suitable for gene therapy. In recent clinical research, adeno-associated virus (AAV) was employed by several teams in the treatment of hemophilia A and B, and the outcomes were encouraging. In this review, we s...

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Bibliographic Details
Main Authors: Xiaolei Pei, Mingzhe Han, Lei Zhang
Format: Article
Language:English
Published: Wolters Kluwer Health 2019-10-01
Series:Blood Science
Online Access:http://journals.lww.com/10.1097/BS9.0000000000000030