Large gene therapy for dystroglycanopathies
| Project/Area Number |
17H06862
|
|---|
| Research Category |
Grant-in-Aid for Research Activity Start-up
|
| Allocation Type | Single-year Grants |
|---|
| Research Field |
Neurology
|
|---|
| Research Institution | Kobe University |
|---|
Principal Investigator |
|
|---|
| Research Collaborator |
Kanagawa Motoi
Toda Tatsushi
|
|---|
| Project Period (FY) |
2017-08-25 – 2019-03-31
|
| Project Status |
Completed (Fiscal Year 2018)
|
| Budget Amount *help |
¥2,730,000 (Direct Cost: ¥2,100,000、Indirect Cost: ¥630,000)
Fiscal Year 2018: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2017: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
|
|---|
| Keywords | 筋ジストロフィー / ジストログリカノパチー / 遺伝子治療 / LARGE |
|---|
| Outline of Final Research Achievements |
Regardless of the causative gene, muscular dystrophies characterized by abnormal glycosylation of dystroglycan are collectively referred to as dystroglycanopathy. We studied whether Large gene therapy is commonly effective to dystrocurinopathy regardless of the causative gene in vivo. We performed Large gene therapy to non-severe dystroglycanopathy model mice in which sugar chains remain a certain extent. As a result, improvement of skeletal muscle necrosis was detected in the non-severe type dystroglycanopathy model mice by Large gene therapy. We found that large gene therapy may be applied to non-severe forms of dystroglycanopathy regardless of causative gene.
|
| Academic Significance and Societal Importance of the Research Achievements |
いずれの病型においても治療法が確立されていないジストログリカノパチーにおいて，新たな治療法の可能性を見出した．
|
Report
(3 results)
Research Products
(2 results)
