ABE8e-NRCH mRNA

ABE8e-NRCH mRNA is a research-stage gene editing reagent consisting of mRNA that encodes an adenine base editor (ABE). It features the ABE8e variant, an evolved adenine deaminase with high catalytic activity, fused to a SpCas9 nickase containing the NRCH PAM-recognition variant. This tool enables precise adenine-to-guanine (A-to-G) conversions in genomic DNA by deaminating adenine to inosine, which is subsequently read as guanine by DNA polymerase. Unlike traditional CRISPR-Cas9 systems, it does not require double-stranded DNA breaks or donor templates, reducing the risk of indels and translocations. The NRCH variant significantly expands the targetable range of the genome by recognizing a broader set of PAM sequences (including NGN) compared to the canonical NGG PAM. It has been primarily used in preclinical research, notably for correcting the HBB-S mutation in hematopoietic stem cells for the treatment of sickle cell disease.

Other names

ABE8e-NRCHABE-8e-NRCHABE 8e-NRCH