Development of site-specific nuclease to control mutated mtDNA in MELAS iPS cell-derived neuronal cells
Project/Area Number |
26860831
|
Research Category |
Grant-in-Aid for Young Scientists (B)
|
Allocation Type | Multi-year Fund |
Research Field |
Pediatrics
|
Research Institution | Fujita Health University |
Principal Investigator |
MATSUMOTO Yuji 藤田保健衛生大学, 医学部, 助教 (50726651)
|
Project Period (FY) |
2014-04-01 – 2016-03-31
|
Project Status |
Completed (Fiscal Year 2015)
|
Budget Amount *help |
¥3,900,000 (Direct Cost: ¥3,000,000、Indirect Cost: ¥900,000)
Fiscal Year 2015: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000)
Fiscal Year 2014: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000)
|
Keywords | MELAS / ミトコンドリアDNA / iPS細胞 |
Outline of Final Research Achievements |
MELAS (Mitochondrial myopathy, encephalopathy, lactic acidosis, and stroke-like episodes), one of mitochondrial diseases, is associated with mitochondrial DNA (mtDNA) mutations. For clinical application, we tried to generate mtDNA-specific TALENs (Transcription activator-like effector nucleases) to control mtDNA heteroplasmy levels. We designed and constructed TALEN plasmids to introduce double strand breaks into mutated or wild-type mtDNA, respectively. The activity and specificity of TALENs were evaluated by SSA (single strand annealing) assay. Wild-type mtDNA-specific TALEN localized in mitochondria decreased mtDNA copy numbers in cell lines.
|
Report
(3 results)
Research Products
(1 results)