2015 Fiscal Year Final Research Report
Development of site-specific nuclease to control mutated mtDNA in MELAS iPS cell-derived neuronal cells
Project/Area Number |
26860831
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Research Category |
Grant-in-Aid for Young Scientists (B)
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Allocation Type | Multi-year Fund |
Research Field |
Pediatrics
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Research Institution | Fujita Health University |
Principal Investigator |
MATSUMOTO Yuji 藤田保健衛生大学, 医学部, 助教 (50726651)
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Project Period (FY) |
2014-04-01 – 2016-03-31
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Keywords | MELAS |
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.
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Free Research Field |
小児科学
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