CorrectSequence’s hemoglobin base editor; plus Denali’s LXR agonist and more

CorrectSequence Therapeutics Co. Ltd. founders and colleagues from multiple Chinese universities created a base editor that completely abolished the interaction of fetal hemoglobin genes with BCL11A, one of the two major repressors responsible for γ-globin gene silencing, with no detectable off-target mutations. Reactivating fetal hemoglobin is a strategy to treat β-hemoglobinopathies.

The authors of the Cell Stem Cell paper identified a region within the fetal hemoglobin HBG1 and HBG2 promoters that, when modified with their previously developed cytosine transformer base editor, triggered the highest level of γ-globin expression compared with other clinical or preclinical editing strategies...