New Prime Editing and non-viral delivery strategies for Gene Therapy

Description

The past few years have witnessed advances in genome editing technologies based on the CRISPR/Cas system. Adapted from the bacterial immune defence mechanisms, the system uses RNA molecules that bind the target sequence and the Cas enzyme to cleave the DNA at the desired location. Currently, delivery of these components into cells is mediated through viral vectors that, however, present a high toxicity risk. Funded by the European Innovation Council, the EdiGenT project introduces a non-viral nanoparticle-based system with minimal side effects for the delivery of gene editing components in cells. Combined with advanced prime editor molecules, it ensures genome modification without the detrimental consequences of double strand DNA breaks.