Svenja Vinke

May 17, 2024 | biorxiv.org | Svenja Vinke |Raphael Ferreira |Michael Moret |Ramiro Martin Perrotta

AbstractAs we enter the era of CRISPR medicines, base editors (BEs) emerged as one of the most promising tools to treat genetic associated diseases. However, unintended bystander editing beyond the target nucleotide poses a challenge to their translation into effective therapies. While many efforts have been made in the design of a universal enzyme with minimal bystander editing, the context dependent activity represents a major challenge for base editing-based therapies.