1999 Fiscal Year Final Research Report Summary
Gene therapy for CNS involvement of Lysosomal storage disease using various viral vector
| Project/Area Number |
10670761
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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 | The Jikei University School of Medicine |
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Principal Investigator |
ITO Fumiyuki Jikei Univ.,Dept.of Pediatrics, asso.prof., 医学部, 助教授 (10057010)
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| Co-Investigator(Kenkyū-buntansha) |
OHASHI Touya Jikei Univ.,Dept.of Pediatrics, assi.prof., 医学部・小児科, 講師 (60160595)
IDA Hiroyuki Jikei Univ.,Dept.of Pediatrics, assi.prof., 医学部・小児科, 講師 (90167255)
ETO Yosikatu Jikei Univ.,Dept.of Pediatrics, prof., 医学部・小児科, 教授 (50056909)
KAWAME Hiroshi Jikei Univ.,Dept.of Pediatrics,senior investigator, 医学部・小児科, 助手 (60246395)
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| Project Period (FY) |
1998 – 1999
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| Keywords | beta-glucuronidase / MPS VII mice / metachromatic leukodystrophy / Gaucher disease |
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| Research Abstract |
We injected adenovirus vector exprssing human beta-glucuronidase into lateral ventricle of MPS VII mice and monitored its expression up to 30 days post ingection. The expression of beta-glucuronidase was restricted in ependmal cells and choroid in ventricles. The mucopolysaccharide storage was reduced in these cell. There was no enzyme competent cells in brain parenchyma. The expression was persisted 30 days.
For gene therapy of Gaucher disease and metachromatic leukodystrophy, we generated adenoviral vector expressing glucocerebrosidase and arylsulfatase A. These vector can restore the enzyme deficiency of fibroblasts from Gauhcer and metachromatic leukodystrophy patients.
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