Custom CRISPR Therapies Could Be Closer Than You Think
Chemical & Engineering News
Rich Horgan still remembers the day his younger brother, Terry Horgan, could no longer summon the strength to walk up the steps of a movie theater. Terry was born with Duchenne muscular dystrophy, a rare genetic disease that causes progressive muscle loss. Today, at 24, he’s too old to participate in one of the many clinical trials of experimental drugs for the rare disease. His heart strength is declining, and he doesn’t sugarcoat his situation: "It sucks, but you got to keep moving."