Therapeutic genome editing of an aberrant splice site in β-thalassemia by CRISPR/Cas9 with multiple sgRNAs.

Autor: Yang F; Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai 200241, China.; BRL Medicine Inc., Shanghai 201108, China., Xu S; Department of Hematology, Qilu Hospital, Cheeloo College of Medicine, Shandong University, Jinan, Shandong 250012, China., Huang C; BRL Medicine Inc., Shanghai 201108, China., Shao Z; BRL Medicine Inc., Shanghai 201108, China., Hu Y; Department of Hematology, Qilu Hospital, Cheeloo College of Medicine, Shandong University, Jinan, Shandong 250012, China., Yang Yang; Department of Hematology, The First Affiliated Hospital of Guangxi Medical University, Nanning, Guangxi 530000, China., Lai Y; Department of Hematology, The First Affiliated Hospital of Guangxi Medical University, Nanning, Guangxi 530000, China., Ke Q; Department of Neurology, The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, Zhejiang 310003, China., Liang D; Department of Obstetrics and Gynecology, The First Affiliated Hospital of Anhui Medical University, Hefei, Anhui 230022, China.; NHC Key Laboratory of Study on Abnormal Gametes and Reproductive Tract (Anhui Medical University), Hefei, Anhui 230032, China., Wu Y; Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai 200241, China.; BRL Medicine Inc., Shanghai 201108, China.
Jazyk: angličtina
Zdroj: Genes & diseases [Genes Dis] 2023 Mar 24; Vol. 11 (1), pp. 15-18. Date of Electronic Publication: 2023 Mar 24 (Print Publication: 2024).
DOI: 10.1016/j.gendis.2023.01.010
Abstrakt: Competing Interests: The authors declare no competing interests.
Databáze: MEDLINE