通过BCL11A直接抑制,控制胎儿对成人血红蛋白的切换

¹Cancer and Blood Disorders Center, Dana Farber Cancer Institute and Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
²Department of Biostatistics and Computational Biology, Dana Farber Cancer Institute and Harvard T.H. Chan School of Public Health, Boston, MA, USA.
³Division of Genetics, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA.
⁴Division of Genetics, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA; Committee on Higher Degrees in Biophysics, Harvard University, Cambridge, MA, USA.
⁵Cell Engineering Division, RIKEN Bioresource Center, Tsukuba, Japan.
⁶Children's Medical Center Research Institute, Department of Pediatrics, University of Texas at Southwestern Medical Center, Dallas, TX, USA.
⁷Division of Genetics, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA; Committee on Higher Degrees in Biophysics, Harvard University, Cambridge, MA, USA; Department of Pathology, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA.
⁸Cancer and Blood Disorders Center, Dana Farber Cancer Institute and Boston Children's Hospital, Harvard Medical School, Boston, MA, USA; Howard Hughes Medical Institute, Boston, MA, USA.

⁰Cancer and Blood Disorders Center, Dana Farber Cancer Institute and Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.

概括

BCL11A直接抑制胎儿血红蛋白 (HbF) 基因表达,导致转换为成人血红蛋白 (HbA). 这种机制涉及γ-环球蛋白促进体中的特定DNA基因,对于调节环球蛋白基因切换至关重要.

关键词:

其他类型切割和运行对DNA进行结合数字基因足迹基因编辑血红蛋白蛋白质结合微阵列镇压指