Prenatal gene editing prevented a lethal metabolic disorder in laboratory mice, suggesting it could be possible to treat congential diseases before a child is born.
Researchers from the Perelman School of Medicine at the University of Pennsylvania and the Children’s Hospital of Philadelphia developed a sophisticated, low-toxicity tool that efficiently edits DNA building blocks in disease-causing genes. The findings were published Monday in the journal Nature Communications.
« Our ultimate goal is to translate the approach used in these proof-of-concept studies to treat severe diseases diagnosed early in pregnancy, » Dr. William H. Peranteau, a pediatric and fetal surgeon in CHOP’s Center for Fetal Diagnosis and Treatment, said in a press release. « We hope to broaden this strategy to intervene prenatally in congenital diseases that currently have no effective treatment for most patients, and result in death or severe complications in infants. »
The scientists used BE3 and joined it with a modified CRISPR-associated protein 9 to form a partially active version of the CRISPR-Cas 9 tool. BE3 was found to be potentially safer than CRISPR-Cas9 because it does not fully cut the DNA molecule and leave it vulnerable to unanticipated errors when the cut is repaired…