OTQ923, a clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9-edited CD34+ hematopoietic stem- and progenitor-cell (HSPC) product, has a targeted disruption of the HBG1 and HBG2 (γ-globin) gene promoters that increases fetal hemoglobin expression in red-cell progeny.
细胞疗法 > 干细胞疗法 > 造血干细胞疗法
Intellia Therapeutics Inc
诺华制药
Intellia Therapeutics Inc
美国