2002 Fiscal Year Final Research Report Summary
INTRAVENOUS GENE THERAPY FOR FAMILIAL HYPERCHOLESTEROLEMIA USING LIGAND-FACILITATED TRANSFER OF A LIPOSOME : LDL RECEPTOR GENE COMPLEX
| Project/Area Number |
13671178
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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 |
Metabolomics
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| Research Institution | KANTO-GAKUIN UNIVERSITY (2002)
Tokyo Medical and Dental University (2001) |
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Principal Investigator |
TANAKA Akira PROFESSOR, COLLEGE OF HUMAN AND ENVIRONMENTAL STUDIES, KANTO-GAKUIN UNIVERSITY, 人間環境学部, 教授 (70171733)
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| Co-Investigator(Kenkyū-buntansha) |
SHICHIRI Masayoshi ASSOCIATE PROFESSOR, DEPARTMENT OF MEDICINE, TOKYO MEDICAL AND DENTAL UNIVERSITY, 医学部, 助教授 (10206097)
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| Project Period (FY) |
2001 – 2002
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| Keywords | GENE THERAPY / WHHL RABBIT / LDL RECEPTOR / RIPOSOME / TRANSFERRIN / LDL RECEPTOR GENE / FAMILIAL HYPERCHOLESTEROLEMIA / TRANSFER OF GENE |
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| Research Abstract |
Familial hypercholesterolemia (FH) is an autosomal dominant disorder because of a mutation in the low-density lipoprotein receptor (LDLR) gene. Although lowering plasma cholesterol decreases the risk of coronary artery disease, FH patients respond poorly to pharmacologic treatment. Transferrin-facilitated intravenous transfer of a cationic liposome rabbit LDLR cDNA complex alleviated hypercholesterolemia in Watanabe Heritable rabbits (WHHL), an animal model of FH. Intravenous treatment does dependently decreased plasma total and LDL cholesterol levels, correlating with an increased level of LDLR mRNA transcripts in leukocytes. Transferrin-facilitated intravenous delivery of cationic liposome LDLR gene complexes could serve as an important adjunct therapy for the treatment of FH.
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