Current Gene Therapy

Ignacio Anegon
Director INSERM UMR 1064-Center for Research in Transplantation and Immunology
CHU de Nantes. 30, boulevard


Adeno-Associated Virus-Mediated Gene Transfer in Hematopoietic Stem/Progenitor Cells as a Therapeutic Tool

Author(s): Li Zhong, Weihong Zhao, Jianqing Wu, Njeri Maina, Zongchao Han, Arun Srivastava.


Hematopoietic stem cells (HSCs) have unique properties of self-renewal, differentiation and proliferation. HSCs are easily accessible, and can be readily delivered back to patients by autologous transplantation, which renders them as attractive targets for ex vivo gene therapy. The adeno-associated virus (AAV) vectors have to date not been associated with any malignant disease, and have gained attention as a potentially safer alternative to the more commonly used retroviral vectors for HSC gene therapy. Although conflicting data exist with regard to HSC transduction by AAV vectors, in this review, we provide an overview of AAV-mediated HSC gene transfer - obstacles as well strategies to improve the transduction efficiency - and the potential use of AAV vectors for gene therapy of human diseases involving HSCs.

Keywords: AAV-mediated transduction, T cell protein tyrosine phosphatase (TC-PTP), FKBP52-KO mice, Host Chromosomal DNA, Hematopoietic Stem cell

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Article Details

Year: 2006
Page: [683 - 698]
Pages: 16
DOI: 10.2174/156652306779010660
Price: $58