Tag: OTQ923

CRISPR-mediated gene activation rescues obesity caused by haploinsufficiency [PreClinical]

CRISPR-Cas9 promoting fetal hemoglobin production improves severe sickle cell disease

byNhat Hung (Benjamin) Lamand1 others

September 13, 2023

1. In this randomized controlled trial, infusion of OTQ923 disrupted inhibitory HBG1 and HBG2 promoter regions of the gamma-globin gene. ...

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