A roadmap to safe, efficient, and stable lentivirus-mediated gene therapy with hematopoietic cell transplantation

作者：Neschadim Anton; McCart J Andrea; Keating Armand; Medin Jeffrey A^*

来源：Biology of Blood and Marrow Transplantation, 2007, 13(12): 1407-1416.

DOI：10.1016/j.bbmt.2007.09.014

• bone marrow
• gene therapy
• hematopoietic stem cell
• lentivirus
• oncoretrovirus
• transplantation

Hematopoietic stem cells comprise a prominent target for gene therapy aimed at treating various genetic and acquired disorders. A number of limitations associated with hematopoietic cell transplantation can be circumvented by the use of cells stably modified by retroviral gene transfer. Oncoretroviral and lentiviral vectors offer means for generating efficient and stable transgene expression. This review summarizes the state of the field today in terms of vector development and clinical experimentation. In particular, concerns with the safety of retroviral vectors intended for clinical gene transfer, applicability of preclinical data in directing clinical trial design, and recent research aimed at resolving some of these issues are addressed. Finally, this review underlines the specific advantages offered by lentiviral gene-transfer vectors for gene therapy in stem cells.

• 出版日期2007-12
• 单位河北医科大学