ABE8e-SpWT base editor

ABE8e-SpWT is an adenine base editor (ABE) consisting of an evolved deoxyadenosine deaminase (ABE8e) fused to a catalytically impaired Streptococcus pyogenes Cas9 (SpCas9) protein, typically a nickase. This CRISPR-based tool is designed to perform precise A•T to G•C base pair conversions without inducing double-strand breaks. In the context of X-linked hyper-IgM syndrome (XHIM), ABE8e-SpWT is utilized to correct pathogenic mutations in the CD40L gene (such as the c.658C>T mutation) in patient-derived hematopoietic stem and progenitor cells (HSPCs) and T cells. By restoring functional CD40L expression, the therapy enables B cell class switching and immune reconstitution, offering a potentially curative approach with improved safety over traditional lentiviral gene addition strategies.

Other names

adenine base editor 8e-SpWTABE8e-SpCas9ABE-8e-SpCas9ABE 8e-SpCas9