Establishment of Modified Retroviral Vector Targeting X-Linked Severe Combined Immunodeficiency
-
- Ling Cai
- Department of Pediatrics, Nippon Medical School
-
- Migita Makoto
- Department of Pediatrics, Nippon Medical School Department of Biochemistry and Molecular Biology, Nippon Medical School Division of Gene Therapy Research Center for Advanced Medical Technology, Nippon Medical School
-
- Hayakawa Jun
- Department of Pediatrics, Nippon Medical School
-
- Fukunaga Yoshitaka
- Department of Pediatrics, Nippon Medical School
この論文をさがす
抄録
Gene therapy targeting hematopoietic stem cells has been proposed as a potential therapy for numerous genetic disorders affecting hematopoiesis. Moloney murine leukemia retroviral vectors are now widely used for clinical gene transfer into hematopoietic progenitors and progeny. However, maintaining expression of therapeutic genes inserted via moloney murine leukemia virus (MoMLV)-based vectors has proven to be more difficult than previously expected. In this study, an MND-IL-2R vector containing IL-2RcγcDNA to treat X-linked severe combined immunodeficiency (X-SCID) was constructed from an MND vector that was modified by substituting the myeloproliferative sarcoma virus (MPSV) enhancer for that of MoMLV, deleting the negative control region located in the long terminal repeat (LTR) as an enhancer, and replacing the primer binding site (PBS) of MoMLV with the PBS of the endogenous murine retrovirus dl587rev. This vector was transduced into human CD34+progenitor cells with comparable efficiency to that of the MoMLV-based vector. The use of this newly created vector may be advantageous for gene therapy of X-SCID.<br>
収録刊行物
-
- 日医大誌
-
日医大誌 71 (1), 51-56, 2004
日本医科大学医学会