1998 Fiscal Year Final Research Report Summary
Stuches of veatment for lysosomal storage diseases in volving central nervous system lesions-model mouse study-
| Project/Area Number |
09470181
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| Research Category |
Grant-in-Aid for Scientific Research (B)
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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 | Osaka University |
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Principal Investigator |
OKADA Shintaro OSAKA University Medical School, Professor, 医学部, 教授 (30028609)
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| Co-Investigator(Kenkyū-buntansha) |
NISHIGAKI Toshinori Osaka University Medical School, Assistant Professor, 医学部, 助手 (20283749)
TANIIKE Masako Osaka University Medical School, Assistant Professor, 医学部, 助手 (30263289)
INUI Koji OSAKA University Medical School, Associate Professor, 医学部, 助教授 (90175208)
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| Project Period (FY) |
1997 – 1998
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| Keywords | Krabbe disease / twitcher mouse / gene therapy / gammaetrovirus vector / Sciatic nerue / galactocerebrosidase |
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| Research Abstract |
The twitcher mouse is an authentic murine model of Krabbe disease, a human genetic demyelinating disease caused by a deficiency of the lysosomal enzyme galacto-cerebrosidase (GALC). The life span of the homozygous mutant, which rarely extends beyond 6 weeks, also provides an excellent model for gene therapy trials of lethal infantile onset single-gene diseases. In this study, recombinant retroviruses were F constructed to transfer human GALC cDNA into twitcher bone marrow cells ex viva. Retrovirally transfeetted bone marrow cells were transplanted intraperitoneally into neonatal twitcher recipients. This treatment improved in body weight curve compared to untreated group and busulfan tread group cspecially at day 26 and 32. This effect is probably due to the increasing GALC activity in sciatic nerves, main foci of twitcher mice, but the survival dates were not different among those groups.
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