ABE7.10

ABE7.10 is a specific adenosine base editor (ABE) developed by David Liu's laboratory at Harvard University and the Broad Institute. It is a CRISPR-based genome editing tool composed of a catalytically impaired Cas9 (typically a nickase, nCas9) fused to an evolved deoxyadenosine deaminase (TadA*). ABE7.10 enables the precise conversion of A•T base pairs to G•C base pairs without inducing double-stranded DNA breaks (DSBs), thereby minimizing indels and chromosomal rearrangements. In the context of hematology, ABE7.10 has been utilized to recreate naturally occurring hereditary persistence of fetal hemoglobin (HPFH) mutations in the γ-globin (HBG1 and HBG2) promoters of hematopoietic stem and progenitor cells (HSPCs). This approach induces fetal hemoglobin (HbF) expression to therapeutic levels, offering a potential treatment for sickle cell disease and β-thalassemia. Beam Therapeutics is the primary commercial developer of this technology, with its clinical candidate Beam-101 utilizing similar base editing principles.

Other names

Adenosine Base Editor 7.10