2002 Fiscal Year Final Research Report Summary
Development of a gene therapy for inherited corneal disease using a lentivirus vector
| Project/Area Number |
13470367
|
|---|
| Research Category |
Grant-in-Aid for Scientific Research (B)
|
| Allocation Type | Single-year Grants |
|---|
| Section | 一般 |
|---|
| Research Field |
Ophthalmology
|
|---|
| Research Institution | Osaka University |
|---|
Principal Investigator |
NISHIDA Koji Department of Ophthalmology, Osaka University Medical School, Assistant Professor, 医学系研究科, 助手 (40244610)
|
|---|
| Co-Investigator(Kenkyū-buntansha) |
TANO Yasuo Department of Ophthalmology, Osaka University Medical School, Professor, 医学系研究科, 教授 (80093433)
WATANABE Hitoshi Department of Ophthalmology, Osaka University Medical School, Associate Professor, 医学系研究科, 助教授 (60252673)
YAMAMOTO Shuji Department of Ophthalmology, Osaka University Medical School, Lectuer, 医学系研究科, 講師 (80294065)
|
|---|
| Project Period (FY) |
2001 – 2002
|
| Keywords | gene therapy / corneal dystrophy / lentivirus vector / cell therapy / gone transfer / knockout mouse / CHST6 / corneal stem cell |
|---|
| Research Abstract |
We investigated a gene therapy for inherited corneal diseases, so-called corneal dystrophies, using a lentivirus vector. Lentivirus vector can transduce non-dividing cells in vitro and in vivo, and mediate efficient transfer and sustained long-term expression of the transgene in varieties of tissues. In this project, we obtained the following results.
(1) We have developed an enhancer-promotor region that drives a cornea-specific expression of a gene of interest.
(2) We have created knockout mice of macular corneal dystrophy using a targeted destruction of CHST6 gene.
(3) We have developed a transplantation approach of corneal epithelial sheets transduced with a lentivirus vector.
(4) We have shown that lentivirus vector can transduce ex vivo corneal endothelial cells. The results suggest promising clinical capabilities for ex vivo gene therapy for the treatment for corneal dystrophies with a lentivirus vector.
|
