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

Affiliations

• 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, Guangxi, 530021, China.
• ⁵ Bioray Laboratories Inc., Shanghai, 200241, China.
• ⁶ Department of Hematology, Xiangya Hospital, Central South University, Changsha, Hunan, 410008, China.
• ⁷ Department of Research and Development, Central Blood Institute, Blood Service Headquarters, Japanese Red Cross Society, Tokyo, 135-8521, Japan.
• ⁸ Cell Engineering Division, RIKEN BioResource Center, Tsukuba, Ibaraki, 305-0074, Japan.
• ⁹ Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai, 200241, China. myliu@bio.ecnu.edu.cn.
• ¹⁰ Department of Hematology, The First Affiliated Hospital of Guangxi Medical University, Nanning, Guangxi, 530021, China. laiyongrong@hotmail.com.
• ¹¹ Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai, 200241, China. dlli@bio.ecnu.edu.cn.