2010 Fiscal Year Final Research Report
Development of novel bone marrow transplantation for treatment of CNS disorders in lysosomal disease.
| Project/Area Number |
20591234
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Single-year Grants |
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| Section | 一般 |
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| Research Field |
Pediatrics
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| Research Institution | Nippon Medical School |
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Principal Investigator |
MIYAKE Noriko Nippon Medical School, 医学部, テクニカルサポートスタッフ (00421206)
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| Co-Investigator(Kenkyū-buntansha) |
MIYAKE Koichi 日本医科大学, 医学部, 准教授 (90267211)
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| Project Period (FY) |
2008 – 2010
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| Keywords | 遺伝 / 先天異常学 / 異染性白質ジストロフィー |
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| Research Abstract |
To evaluate the contribution of bone marrow (BM) cells to treat neurological disorders, we examined the effectiveness of BM cells expressing the homeobox B4 (HoxB4) gene to cure mice with metachromatic leukodystrophy (MLD) through transplantation. Increased number of donor cells was observed in brains of the MLD mice transplanted with HoxB4-transduced BM cells (B4MLD) in contrast to those transplanted with control GFP-transduced BM cells (MIGMLD). Transplantation of BM cells overexpressing HoxB4 appears to effectively prevent the progression of MLD in this mouse model. These findings support the idea that hematopoietic stem cells (HSCs) transduced with a HoxB4 expression vector could be useful carriers of therapeutic proteins into the brain for regeneration of oligodendrocytes to treat such demyelinating disorders as MLD, Krabbe disease and multiple sclerosis.
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