Reactivation of γ-globin expression through Cas9 or base editor to treat β-hemoglobinopathies

查看全文 作　　者：Liren [1]Wang;Linxi [1]Li;Yanlin [2,3]Ma;Handan [1]Hu;Qi [2,3]Li;Yang [4]Yang;Wenbang [1]Liu;Shuming [1]Yin;Wei [5]Li;Bin [6]Fu;Ryo [7]Kurita;Yukio [8]Nakamura;Mingyao [1]Liu;Yongrong [4]Lai;Dali [1]Li

机构地区：[1]Shanghai Key Laboratory of Regulatory Biology,Institute of Biomedical Sciences and School of Life Sciences,East China Normal University,Shanghai 200241,China;[2]Hainan Provincial Key Laboratory for human reproductive medicine and Genetic Research,Hainan Provincial Clinical Research Center for Thalassemia,The First Affiliated Hospital of Hainan Medical University,Hainan Medical University,Haikou,Hainan 570102,China;[3]Key Laboratory of Tropical Translational Medicine of Ministry of Education,Hainan Medical University,Haikou,Hainan 571199,China;[4]Department of Hematology,The First Affiliated Hospital of Guangxi Medical University,Nanning,Guongxi 530027,China;[5]Bioray Laboratories Inc.,Shanghai 200241,China;[6]Deportment of Hematology,Xiangya Hospital,Central South University,Changsha,Hunon 410008,China;[7]Deportment of Research and Development,Central Blood Institute,Blood Service Headquarters,Japanese Red Cross Society,Tokyo 735-8521,Japan;[8]Cell Engineering Division,RIKEN BioResource Center,Tsukuba,Ibaroki 305-0074,Japan

出　　处：《Cell Research》索引2020年第30卷第3期,共3页

基　　金：partially supported by grants from the National Key R&D Program of China(2019YFA0110802,2019YFA0802800);Major Science and Technology Program of Hainan Province(ZDKJ2017007);the National Natural Science Foundation of China(81670470,81873685);grants from the Shanghai Municipal Commission for Science and Technology(18411953500);the Innovation program of Shanghai Municipal Education Commission(2019-01-07-00-05-E00054);the Fundamental Research Funds for the Central Universities. 摘　　要：Dear Editor,Mutations in the β-globin gene,the essential component of adult hemoglobin(HbA;a2p2),results in either a production of aberrant sickle hemoglobin(HbS)leading to sickle cell disease(SCD)or an insufficient β-globin synthesis leading to β-thalassemia.These two major forms of β-hemoglobinopathies cause impaired erythropoiesis and life-threatening anemia.Clinical evidence has suggested that reaaivation of fetal γ-globin(HBG)gene expression which is normally silenced after birth by certain genetic mutations can ameliorate the clinical course of β-hemoglobinopathies. 关键词：IMPAIRED FETAL ANEMIA

相关文献

参考文献（12）
引证文献（20）
耦合文献（0）

参考文献(12)

引证文献(20)

互联网出版许可证新出网证(渝)字10号全国400电话 - 免长途话费

Reactivation of γ-globin expression through Cas9 or base editor to treat β-hemoglobinopathies

参考文献(12)

引证文献(20)

用户提示