Feature Genetics

Most of today’s gene therapies rely on viruses — and that’s a problem

CRISPR and new approaches to delivery may help treat rare diseases

Will Ungerer lies in a hospital bed, next to a teddy bear, as doctor Jerry Mendell leans over him. Both are looking at the camera and smiling.

In 2018, Will Ungerer, shown with doctor Jerry Mendell at Nationwide Children’s Hospital in Columbus, Ohio, received experimental gene therapy for Duchenne muscular dystrophy, a disease that breaks down muscle.

S. UNGERER

By Tina Hesman Saey

Will Ungerer isn’t a typical fourth-grader.

The 10-year-old from Midlothian, Va., plays tag with his friends and swims in the ocean.