Thursday, March 21, 2019
Guided by CRISPR, prenatal gene editing shows proof-of-concept in treating disease before birth
In an article from Science Daily, scientists conducted gene editing to prevent a lethal liver disease in laboratory animals and offers to treat human congenital disease before birth. Researchers from Children's Hospital of Philadelphia used low-toxic DNA base editing tools to turn off the effects of a disease causing genetic mutation. In this study, scientists performed prenatal gene editing to improve liver function and to prevent neonatal death in a group of mice that had been engineered with a mutation causing the lethal disease called hereditary tyrosinemia type 1 (HT1). In humans, HT1 usually occurs during infancy and treated with a medicine called nitisinone and strict diet. But, when the treatment fails the patients are in severe risk of liver failure or cancer. Scientists suggested that prenatal gene editing can be used to prevent disease like HT1 and many other congenital diseases. The research used base editor 3 (BE3) and a modified CRISPR associated with protein 9 (CRISPR-Cas 9) tool to carry an enzyme to a particular genetic location in the liver cells of the fetal mice. The enzyme modified the targeted genetic sequence of liver cells chemically by changing the type of DNA bases into another. As a result, the mice showed reduced level of cholesterol and had improved liver function.
A future application for DNA base editing could be correcting disease-causing mutations and to improve functions of organs beyond liver. I think this technique would be very useful for doctors to treat diseases during early pregnancy to ensure the health of the fetus.