ARTICLE | Translation in Brief
Risking CRISPR correction before birth
CHOP-UPenn team leaves open to question risks of CRISPR gene editing in utero
April 25, 2019 5:40 PM UTC
A team from CHOP and UPenn has published its second study showing CRISPR-based gene editing can treat deadly diseases in the womb but has yet to address the safety repercussions for the mother and fetus.
In a study published this month in Science Translational Medicine, researchers at Children's Hospital of Philadelphia and the University of Pennsylvania used a CRISPR-Cas9 system in prenatal mice to edit a SP-C mutant that causes lethal lung disease. The system, delivered with a viral vector into the amniotic fluid, decreased toxic levels of the protein and increased survival after birth...