GENOME EDITING OF A HUMAN EMBRYO – COULD CORRECTION REPLACE SCREENING?

Abstract

Genome editing (CRISPR-Cas9 or Base Editing) is a revolutionary tool which allows DNA or RNA to be altered at directed loci in the genome with ease and efficiency. The technology, which is derived from and mimics the immune system of bacteria, offers unprecedented ability to alter, correct or insert genes into a genome. The technology has been adopted globally in the research setting and offers limitless potential for personalised medicine; indeed, somatic cell therapies are already benefiting from this technology. The advances in genome editing coupled with assisted reproductive technologies and genomics lend themselves to germline therapy and the correction of genetic disorders in embryos. Could correction replace screening? In order for this to reach the clinic, significant and rigorous testing must be carried out ensure that edits are controllable, reproducible and that mosaicism is avoided. Our research focuses on the delivery of components of CRISPR, assessing the accuracy of edits and measuring mosaicism in edited murine preimplantation embryos.