Regenerative therapy using gene-edited iPS cells derived from an epidermolysis bullosa patient
| Project/Area Number |
17H05089
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| Research Category |
Grant-in-Aid for Young Scientists (A)
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| Allocation Type | Single-year Grants |
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| Research Field |
Dermatology
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| Research Institution | Niigata University |
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Principal Investigator |
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| Project Period (FY) |
2017-04-01 – 2020-03-31
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| Project Status |
Completed (Fiscal Year 2019)
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| Budget Amount *help |
¥18,460,000 (Direct Cost: ¥14,200,000、Indirect Cost: ¥4,260,000)
Fiscal Year 2019: ¥5,330,000 (Direct Cost: ¥4,100,000、Indirect Cost: ¥1,230,000)
Fiscal Year 2018: ¥5,720,000 (Direct Cost: ¥4,400,000、Indirect Cost: ¥1,320,000)
Fiscal Year 2017: ¥7,410,000 (Direct Cost: ¥5,700,000、Indirect Cost: ¥1,710,000)
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| Keywords | 表皮水疱症 / reframing治療 / 遺伝子治療 / iPS細胞 / 遺伝子編集 / フレームシフト変異 / 再生医療 / CRISPR/Cas9 / 皮膚科学 |
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| Outline of Final Research Achievements |
Recessive dystrophic epidermolysis bullosa is an inherited disorder characterized by blisters and erosions on the whole body. It was considered that a radical treatment could be developed by combining a local treatment in which fibroblasts and epidermal keratinocytes differentiated from iPS cells genetically treated using gene editing technology such as CRISPR/Cas9 are directly administered to the affected area and a systemic treatment by systemic administration of hematopoietic/mesenchymal stem cells. In this study, we established iPS cells from epidermal keratinocytes derived from patients with epidermolysis bullosa, and confirmed the possibility of differentiation into mesenchymal stem cells. Furthermore, we have established a technology to specifically and efficiently repair gene mutations that are frequently found in Japanese patients.
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| Academic Significance and Societal Importance of the Research Achievements |
生体において免疫は他人の細胞を排除するように働くため、再生医療には自己の細胞を用いる必要がある。遺伝性疾患では、患者細胞には遺伝子変異があるため、患者の細胞に遺伝子治療を施して、再度患者自身に戻す必要がある。この治療法を実用化するためには、①細胞を必要分増殖させること、また②遺伝子治療した細胞を全身に行き渡らせる必要があった。本研究では、患者由来細胞の遺伝子治療法を確立し、さらに自己増殖が可能なiPS細胞を皮膚細胞や全身に遊走することができる細胞に分化させることに成功し、遺伝性皮膚疾患の遺伝子・再生医療に向けた実証実験を行った。
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Report
(4 results)
Research Products
(14 results)
