Patients Unsure about the Value of Cutting-Edge Gene-Editing Technology

The revolutionary gene-editing technology poised to reshape how researchers attack and prevent disease yesterday received a lukewarm reception from patient groups. Representatives from several patient advocacy organizations gathered in Washington, D.C., at a public meeting on gene editing to discuss if they would want researchers to one day tap this technology—first in the laboratory but eventually in the clinic—in an effort to prevent or treat serious inherited maladies including muscular dystrophy, cystic fibrosis and sickle-cell disease.

“We are not one advocacy or affected community,” said Sharon Terry, the president and CEO of Genetic Alliance and a member of the  National Academies of Sciences, Engineering and Medicine panel that organized the meeting. Terry pointed to an earlier survey she conducted among more than 1,000 people in the patient community that pointed to human gene-editing opinions ranging from “What is gene editing?” to “Hell yes!”

Patients and their families are wrestling with these questions because new, powerful gene-editing techniques such as CRISPR–Cas9 allow researchers to make targeted changes in DNA much more easily than ever before. Such deletions or...