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

作　　者：Liren Wang Linxi Li Yanlin Ma Handan Hu Qi Li Yang Yang Wenbang Liu Shuming Yin Wei Li Bin Fu Ryo Kurita Yukio Nakamura Mingyao Liu Yongrong Lai Dali 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》2020年第3期276-278,共3页细胞研究（英文版）

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 

分 类 号：Q51[生物学—生物化学]