Gene editing could help treat lethal diseases before birth
| Updated: Oct 10, 2018, 10:47 IST
Scientists have performed prenatal gene editing to prevent a metabolic disorder in animals and this they claim might help treat human congenital diseases before birth. Using both CRISPR-Cas9 and base editor 3 (BE3) gene-editing tools, the researchers from the University of Pennsylvania and Children's Hospital of Philadelphia (CHOP) in the US reduced cholesterol levels in healthy mice treated in utero by targeting a gene that regulates those levels.
"Our ultimate goal is to translate the approach used in these proof-of-concept studies to treat severe diseases diagnosed early in pregnancy," said William H Peranteau, a pediatric and foetal surgeon in CHOP's Center for Fetal Diagnosis and Treatment. "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," said Peranteau. "We used base editing to turn off the effects of a disease-causing genetic mutation," said Kiran Musunuru, an associate professor at University of Pennsylvania. "We also plan to use the same base-editing technique not just to disrupt a mutation's effects, but to directly correct the mutation," said Musunuru.
"Our ultimate goal is to translate the approach used in these proof-of-concept studies to treat severe diseases diagnosed early in pregnancy," said William H Peranteau, a pediatric and foetal surgeon in CHOP's Center for Fetal Diagnosis and Treatment. "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," said Peranteau. "We used base editing to turn off the effects of a disease-causing genetic mutation," said Kiran Musunuru, an associate professor at University of Pennsylvania. "We also plan to use the same base-editing technique not just to disrupt a mutation's effects, but to directly correct the mutation," said Musunuru.