2013 Fiscal Year Final Research Report
Therapeutical strategy of Spinal muscular atrophy by RNA virus vector delivery to human iPS-derived neural cells
| Project/Area Number |
23659530
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| Research Category |
Grant-in-Aid for Challenging Exploratory Research
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| Allocation Type | Multi-year Fund |
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| Research Field |
Pediatrics
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| Research Institution | Microbial Chemistry Research Foundation |
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Principal Investigator |
ARAKAWA Masayuki 公益財団法人微生物化学研究会, 微生物化学研究所, 研究員 (90398868)
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| Co-Investigator(Renkei-kenkyūsha) |
SAITO Kayoko 東京女子医科大学, 医学部, 教授 (90138834)
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| Project Period (FY) |
2011 – 2013
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| Keywords | 脊髄性筋萎縮症 / ポリオウイルス / 運動神経細胞 / 人工多能性幹細胞 |
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| Research Abstract |
Spinal muscular atrophy (SMA), a genetic neuromuscular disorder, leads to motor neuron degeneration. SMA is caused by reduced production of the survival of motor neuron (SMN) protein resulting from the homozygous deletion or mutation of the SMN1 gene. To date, however, it remains unclear how SMN protein deficiency causes the selective spinal motor neuron death seen in SMA because of a lack of a method to express SMN proteins by the appropriate delivery to the spinal cord. Therefore, an effective treatment of SMA does not presently exist. This study was focused on the potential of a neurotropic RNA virus, poliovirus (PV)-based vector as a novel therapeutic delivery system of exogenous protein in human iPS-derived neural cells and SMA-derived cells.In this study, PV-based expression vector delivery method exhibited an insight into the GFP or GFP-SMN protein expression during an early stage of human iPS-derived neural cells and SMA patient-derived cells.
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