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...
Main Authors: | , , |
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Format: | Article |
Language: | English |
Published: |
Wolters Kluwer Health
2019-10-01
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Series: | Blood Science |
Online Access: | http://journals.lww.com/10.1097/BS9.0000000000000030 |