2015 Fiscal Year Final Research Report
Purification of rAAV-1 and -9 with ultracentrifugation-free technique towards GMP production.
| Project/Area Number |
25460407
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Multi-year Fund |
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| Section | 一般 |
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| Research Field |
Human genetics
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| Research Institution | Nippon Medical School |
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Principal Investigator |
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| Project Period (FY) |
2013-04-01 – 2016-03-31
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| Keywords | AAV ベクター精製法 / 適正製造規範準拠 / down-stream processing / AAV type 1 vector / AAV type 9 vector |
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| Outline of Final Research Achievements |
The current production of rAAV from the transfected cell lysate and purification based on CsCl or iodixanol density ultracentrifugation are not suitable for large-scale processing. Although rAAV1 and rAAV9 are promising therapeutic vectors for genetic neuromuscular disorders, the large-scale purification method for those vectors has not yet been established. In this study, we elaborate the novel chromatography-mediated methods for purification of rAAV1 and rAAV9 from the serum-free culture supernatant with ultracentrifugation-free technique towards large-scale and GMP production.
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| Free Research Field |
生化学・分子生物学
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