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Genetic Linkage

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

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 patients with a form of retinal blindness (the basis of The Forever Fix), while Zolgemsa treats spinal muscular atrophy, a disease typically lethal in young children.

 

The latest in a series of setbacks, beginning in 1999 with the death of 18-year-old Jesse Gelsinger, came just yesterday. The FDA placed a clinical hold on two gene therapy trials for sickle cell disease, following reports of blood cancer in two trial participants. But that's not all.

 

To continue reading, go to DNA Science, where this post first appeared.

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Should Gene Doping Studies Be Published?

In late 2011, creation of a lab strain of of H5N1 influenza capable of spreading easily among ferrets, and presumably us, sparked heated debate about whether and when to publish scientific research that could do harm. The same could be said for gene doping.

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