| Project/Area Number |
26461281
|
|---|
| Research Category |
Grant-in-Aid for Scientific Research (C)
|
| Allocation Type | Multi-year Fund |
|---|
| Section | 一般 |
|---|
| Research Field |
Neurology
|
|---|
| Research Institution | Teikyo University |
|---|
Principal Investigator |
|
|---|
| Co-Investigator(Kenkyū-buntansha) |
真先 敏弘 帝京科学大学, 医療科学部, 教授 (00585028)
萩原 宏毅 帝京科学大学, 医療科学部, 教授 (80276732)
|
|---|
| Co-Investigator(Renkei-kenkyūsha) |
Matsumura Kiichiro 帝京大学, 医学部, 教授 (50260922)
|
|---|
| Project Period (FY) |
2014-04-01 – 2017-03-31
|
| Project Status |
Completed (Fiscal Year 2016)
|
| Budget Amount *help |
¥4,810,000 (Direct Cost: ¥3,700,000、Indirect Cost: ¥1,110,000)
Fiscal Year 2016: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2015: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2014: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000)
|
|---|
| Keywords | 筋ジストロフィー / α-ジストログリカノパチー / 福山型先天性筋ジストロフィー / 糖転移酵素 / 酵素補充療法 / α-ジストログリカン / フクチン |
|---|
| Outline of Final Research Achievements |
α-Dystroglycan (α-DG) stabilizes plasma membrane by binding with laminin via its glycan chain.
Mutations of glycpsyltransferases involved in the glycosylation ofα-DG lead to multi-organ disorders including muscular dystrophy, brain anomaly and eye abnormality, which is called α-dystroglycanopathy. In this study, we conducted basic research to develop glycosyltransferase replacement therapy forα-dystroglycanopathy, which aims to treat the patients by correcting abnormal glycan structure of α-DG with glycpsyltransferases protein. Further, using CRISPR/Cas9 genome editing technology, we established cell culture assay system for the glycosyltransferase replacement therapy, and advocated to use ricin-B subunit as a trafficking tag to the endoplasmic reticurum.
|
