Enhanced fetal hemoglobin production via dual-beneficial mutation editing of the HBG promoter in hematopoietic stem and progenitor cells for β-hemoglobinopathies
通过对造血干细胞和祖细胞中的 HBG 启动子进行双重有益突变编辑,增强胎儿血红蛋白的生成,用于治疗 β-血红蛋白病
期刊:Stem Cell Research & Therapy
影响因子:7.1
doi:10.1186/s13287-024-04117-0.
Prathibha Babu Chandraprabha #,Manoj Kumar K Azhagiri #,Vigneshwaran Venkatesan,Wendy Magis,Kirti Prasad,Sevanthy Suresh,Aswin Anand Pai,Srujan Marepally,Alok Srivastava,Kumarasamypet Murugesan Mohankumar,David I K Martin,Saravanabhavan Thangavel