Researchers can help to shepherd the new drugs to market

Sonam Ghosh and his collaborators, who were working 10-to-12-hour days, were working on a one-off treatment that would edit the DNA in the brain cells of 20-year-old Uditi Sarafs. The team genetically engineered the woman’s FENIB mutation into cells grown in the lab. Uditi had Neuroserpin clumps in some of her neurons and the team was designing a one-off treatment to stop them.