For the first time, scientists have performed prenatal gene editing to prevent a lethal metabolic disorder in laboratory animals, offering the potential to treat human congenital diseases before birth. Research published in Nature Medicine from the Perelman School of Medicine and the Children’s Hospital of Philadelphia offers proof-of-concept for prenatal use of a sophisticated, low-toxicity tool that efficiently edits DNA building blocks in disease-causing genes.
Using both CRISPR-Cas9 and base editor 3 (BE3) gene-editing tools, the team reduced cholesterol levels in healthy mice treated in utero by targeting a gene that regulates those levels. They also used prenatal gene editing to improve liver function and prevent neonatal death in a subgroup of mice that had been engineered with a mutation causing a lethal liver disease.
“We used base editing to turn off the effects of a disease-causing genetic mutation,” says study co-leader Kiran Musunuru, an associate professor of cardiovascular medicine. “We also plan to use the same base-editing technique not just to disrupt a mutation’s effects, but to directly correct the mutation.”
Read more at Penn Medicine News.