Current trends in gene recovery mediated by the CRISPR-Cas system

Abstract

Experimental & Molecular Medicine: Genetic disease: Gene editing for recovery The CRISPR-Cas gene editing system, which relies on small RNA molecules to guide a gene-editing enzyme to specific locations on DNA, is being developed as an effective tool for correcting genetic disorders. Researchers in South Korea led by Sangsu Bae at Hanyang University in South Korea, review recent progress towards such “gene recovery” procedures. The possibilities range from correcting mutations at the level of a single base in the base sequence of DNA, to deleting, inverting or inserting large sections of DNA to correct major structural abnormalities. The authors discuss the pros and cons of different procedures, including CRISPR-Cas nucleases, base editors, and prime editors. They expect current laboratory animal investigations will lead to a new era in human genetic medicine, yielding treatments for genetic diseases that cannot currently be treated with drugs.