1996 Fiscal Year Final Research Report Summary
ESTABLISHMENT OF A RAT STRATIN WITH MUCOPOLYSUCCHARIDOSIS TYPE VI AS AN ANIMAL MODEL FOR GENE THERAPY
| Project/Area Number |
07558240
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| Research Category |
Grant-in-Aid for Scientific Research (A)
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| Allocation Type | Single-year Grants |
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| Section | 試験 |
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| Research Field |
Laboratory animal science
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| Research Institution | OKAYAMA UNIVERSITY |
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Principal Investigator |
KUNIEDA Tetsuo OKAYAMA UNIVERSITY,FACULTY OF AGRICULTURE,ASSOCIATE PROFESSOR, 農学部, 助教授 (80178011)
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| Co-Investigator(Kenkyū-buntansha) |
AMAO Hiromi NIPPON VETERINARY AND AMINAL SCIENCE UNIVIERSITY,DEPARTMENT OF LABOLATORY ANIMAL, 獣医畜産学部, 講師 (60151114)
WAKAFUJI Yasumasa IMAMICHI INSTITUTE FOR ANIMAL REPRODUCTION., 副主任研究員 (80158592)
YOSHIDA Midori SASAKI INSTITUTE,DEPARTMENT OF PATHOLOGY., 病理部, 研究助手 (70201861)
SAKIYAMA Takeshi ST MARIANNA UNIVERSITY SCHOOL OF MEDICINE,FIRST DEPARTMENT OF PATHOLOGY,ASSOCIAT, 医学部, 助教授 (20130510)
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| Project Period (FY) |
1995 – 1996
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| Keywords | RATS / ANIMAL MODEL / MUCOPOLYSUCCHARIDOSIS / LYSOSOME / ARYLSULFATASE / MUTATION / SOMATIC-CELL HYBRIDS / MAPPING |
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| Research Abstract |
The present research project was carried out to establish the MPR rat as an animal model for human hereditary mucopolysuccharidosis type VI by identifying the mutation responsible for the phenotypes and by examinating therapeutic effects of bone marrow transplantation.
We compaired the nucleotide sequences of the rat arylsulfatase B gene by cloning nad sequencing the gene, and found an insertional mutation that causes a fram-shift of the gene. We further revealed that an amplification of the region including the mutation enables to easily identify carriar rats of the mutation.
We performed bone marrow transplantation to confirm therapeutic effects of supplement of the normal enzyme producing cells. The recipient rats showed no accumulation of glycosaminoglycan in organs and no urinary excretion of the glycosaminoglycan but partial resoluation of facial dysmorphia and bony feutures, indicating therapeutic effects of the bone marrow transplantation.
We, therefore, concluded that the MPR rat is a useful animal model for mucopolysaccharidosis type VI of human.
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[Publications] Kunieda, T., Simonaro, C.M., Yoshida, M., Ikadai, H., Levan, G., Desnick, R.J., and Schuchman, E.H.: "Mucopolysaccharidosis type VI in rats : Isolation of cDNA encoding arylsulfataseB,chromosomal localizaiton of the gene, and identification of the mutation." Genomics. 29. 582-587 (1995)
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[Publications] Kunieda, T., Kumagai, T., Tsuji, T., Ozaki, T., Karaki, H., and Ikadai, H.: "A mutation in endothelin-B receptor gene causes myenteric aganglionosis and coat color spotting in rats." DNA Res.3. 101-105 (1996)
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[Publications] Karaki, H., Mitui-Saito, M., Takimoto M., Oda, K., Okada, T., Ozaki, T., and Kunieda, T.: "Lack of endothelin ETB receptor binding and funciton in the rat with a mutant ETB receptor gene." Biochem.Biophys.Res.Commun.222. 139-143 (1996)
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[Publications] Kobayashi, E., Tachibana, M., Ikadai, H., and kunieda, T.: "Localization of Na^+, K^+-ATPase a 2 subunit gene, ATP1A2, on rat chromosome 13." Mamm.Genom. 6. 889 (1995)
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