ABE8e ribonucleoprotein

ABE8e ribonucleoprotein (RNP) is a CRISPR-based gene editing therapeutic consisting of the ABE8e adenine base editor protein complexed with guide RNAs. ABE8e is an evolved adenine base editor that enables precise A-to-G (adenine to guanine) base pair conversions in DNA without creating double-strand breaks or requiring donor DNA templates. The ABE8e protein contains eight mutations in the TadA deaminase domain that significantly enhance its editing efficiency compared to earlier versions like ABE7.10. The therapeutic is typically delivered as a ribonucleoprotein complex via lipid nanoparticles (LNPs), electroporation, or experimental image-guided methods such as focused ultrasound with microbubbles. It is being investigated for the treatment of multiple genetic diseases, including β-thalassemia (by targeting the BCL11A enhancer and HBG promoters to induce fetal hemoglobin), aniridia (by correcting PAX6 gene variants), and dystrophic epidermolysis bullosa (by correcting COL7A1 mutations).

Other names

ABE8e adenine base editor ribonucleoproteinABE-8e adenine base editor ribonucleoproteinABE 8e adenine base editor ribonucleoprotein