Determined Parents Are Moving the Needle on Gene Therapy

March 21, 2017

(MIT Technology Review) – Frase is very likely the reason why the same treatment is now about to be tested in humans. In recent years, gene therapies have become safer and better at hitting their intended targets in the body, leading to a handful of remarkable cures in clinical trials. Advocates for rare-disease patients—especially determined parents like Frase—are increasingly seeking to start gene-therapy programs. They are establishing patient advocacy organizations, raising money for research, and even founding their own biotechnology startups to find treatments where few or none currently exist.