New Technique Improves Gene Editing Efficiency While Reducing DNA Deletion Sizes

Experts at Wake Forest Institute for Regenerative Medicine (WFIRM) successfully developed a technique to improve the efficiency of gene editing using CRISPR-Cas9 while decreasing DNA deletion sizes. This technique, reported in Nucleic Acids Research, is a vital step in the development of gene therapies for the treatment of genetic diseases.

CRISPR-Cas9 is the most used gene editing technique and has a wide range of potential applications. While CRISPR-Cas9 mainly generates short insertions or deletions at a target site, large DNA deletions can also occur. Such large deletions can cause safety concerns and may reduce functional editing efficiency. The research team looked for ways to address the occurrence of unpredictable long DNA deletions in the target site and developed a technique to protect against them.

“This technique increased genome editing efficiency in primary cells and did not increase DNA substitution rates or off-target rates. It also decreased large deletions, thus increasing safety. We can increase the percentage of desirable types of mutations. This improves the efficiency of disrupting disease-causing genes or restoring disrupted genes," said Anthony Atala, WFIRM Director and one of the authors of the paper.

Read more from News-Medical.Net and Nucleic Acids Research.