T1ABE

T1ABE (Type I CRISPR adenine base editor) is an innovative gene-editing platform developed to address genetic mutations by performing precise single-nucleotide substitutions. Unlike conventional Cas9-based editors, T1ABE utilizes a Type I CRISPR system which provides a "sliding-window" capability, allowing the editing window to be tuned across a 40-nucleotide region by adjusting the length of the guide RNA. This flexibility is particularly useful for targeting sites like the CFTR-G542X mutation in cystic fibrosis, where suitable Protospacer Adjacent Motifs (PAMs) for Cas9 are absent. The system incorporates TadA-8e deaminase variants to convert A•T base pairs to G•C, and research has demonstrated its ability to restore CFTR protein expression and channel function in human bronchial epithelial cell models.

Other names

Type I CRISPR adenine base editor