Submit Manuscript  

Article Details


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

[ Vol. 6 , Issue. 6 ]

Author(s):

Li Zhong, Weihong Zhao, Jianqing Wu, Njeri Maina, Zongchao Han and Arun Srivastava   Pages 683 - 698 ( 16 )

Abstract:


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

Affiliation:

Division of Cellular and Molecular Therapy, Cancer and Genetics Research Complex, Room 492-A, University of Florida College of Medicine, PO Box 103633, Gainesville, FL 32610-3633, USA.



Read Full-Text article