Search-and-replace genome editing without double-strand breaks or donor DNA.

ABSTRACT: Most genetic variants that contribute to disease¹ are challenging to correct efficiently and without excess byproducts^2-5. Here we describe prime editing, a versatile and precise genome editing method that directly writes new genetic information into a specified DNA site using a catalytically impaired Cas9 endonuclease fused to an engineered reverse transcriptase, programmed with a prime editing guide RNA (pegRNA) that both specifies the target site and encodes the desired edit. We performed more than 175 edits in human cells, including targeted insertions, deletions, and all 12 types of point mutation, without requiring double-strand breaks or donor DNA templates. We used prime editing in human cells to correct, efficiently and with few byproducts, the primary geneti

SUBMITTER: Anzalone AV 

PROVIDER: S-EPMC6907074 | biostudies-literature | 2019 Dec

REPOSITORIES: biostudies-literature