BE3-gMH

BE3-gMH is an experimental in vivo **CRISPR cytosine base-editing** intervention comprising base editor 3 and the gMH guide RNA. Delivered to mouse liver using an adenoviral vector, it directs C-to-T base conversion at the PCSK9 locus near codon W159, generating predominantly nonsense loss-of-function alleles in human PCSK9 and mouse Pcsk9 without creating intended DNA double-strand breaks. In a human-PCSK9 knock-in hypercholesterolemic mouse model, it reduced circulating PCSK9 and total cholesterol; it remains a preclinical research construct with no assigned INN or marketed product. ([link.springer.com](https://link.springer.com/content/pdf/10.1186/s12915-018-0624-2.pdf))

Other names

BE3-gMHBE-3-gMHBE 3-gMH