2024-03-28T18:10:43Z
https://niigata-u.repo.nii.ac.jp/oai
oai:niigata-u.repo.nii.ac.jp:00017964
2022-12-15T03:49:16Z
453:456
471:537:538:1127
Gene Therapy for Sickle Cell Disease (Gene Therapy : Advances in Research and Treatment)
4) 鎌状貧血の遺伝子治療(シンポジウム 遺伝子治療をめぐる諸問題, 第509回新潟医学会)
4) 鎌状貧血の遺伝子治療(シンポジウム 遺伝子治療をめぐる諸問題, 第509回新潟医学会)
古川, 達雄
112777
gene therapy
Sickle cell disease
LCR
遺伝子治療
鎌状貧血
Sickle cell disease is the one of the molecular disease with a point mutation at the 6th amino in β globin gene. This disease is life threatening because of the obstruction of micro-circulation. Although there are some therapeutic ways for the disease, it is difficult for the patients to obtain long term survival. A lot of patients are waiting for developing a new therapy such as a gene therapy. There are several ways for gene therapy of sickle cell disease, we thought that the expression of γ-globin gene at adult stage might be the best way for the therapy. We have developed the backbone of the vector we would try to use for the gene therapy. The vector may consist; (1) insulator at the both sides, (2) globin gene with the promoter, (3) a strong enhancer such as the LCR (Locus Control Region), (4) the gene element for the in vivo selection, such as a drug resistance gene.
departmental bulletin paper
新潟医学会
1996-08
application/pdf
新潟医学会雑誌
8
110
316
318
新潟医学会雑誌
AN00182415
00290440
https://niigata-u.repo.nii.ac.jp/record/17964/files/110(8)_316-318.pdf
jpn