Research

Rare Disease Day 2021: Gene Therapy Ups and Downs, Again

1 month ago   |   By DNA Science

I can't believe it's been a decade since I researched and wrote The Forever Fix: Gene Therapy and the Boy Who Saved It. Since then I've shared stories of other families doing amazing things to help researchers develop treatments for their loved ones' rare diseases. The need is all the more compelling in these days of the pandemic .
Now entering its fourth decade, gene therapy continues along what seems at times a never-ending rocky road , riding the waves of fantastic success and plunging setbacks.
A Slow Start
The US has approved just two gene therapies. Luxturna has provided vision to...
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