Researchers funded by NHLBI have performed prenatal gene editing to prevent a lethal congenital metabolic disease in mice. The study findings, published in Nature Medicine, offer proof of concept for the possibility of genetic therapies before birth.
The researchers used CRISPR, the gene editing tool, to alter the DNA of laboratory mice still in the womb. They 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 other mice that had a mutation that causes the often fatal liver disease hereditary tyrosinemia type 1 (HT1).
“A significant amount of work needs to be done before prenatal gene editing can be translated to the clinic, including investigations into more clinically relevant delivery mechanisms and ensuring the safety of this approach,” study co-leader William H. Peranteau, MD, said in a press release. Peranteau is a pediatric and fetal surgeon in Children's Hospital of Philadelphia’s Center for Fetal Diagnosis and Treatment. . “Nonetheless, we are excited about the potential of this approach to treat genetic diseases of the liver and other organs for which few therapeutic options exist,” Peranteau added.