Viewpoint: Experimental gene therapy saved my sons’ eyesight

Anthony and his brother Nicholas, both who have LCA. Image: Kristin Papiro

Not long after we brought my newborn son, Anthony, home from the hospital, we noticed his eyes kept darting to the nearest light. If left in a room alone, he couldn’t self-soothe unless we placed him beside a sunlit window out of which he would obsessively gaze. He was eventually diagnosed with Leber congenital amaurosis, or LCA, a rare retinal disease affecting one out of every 50,000 newborns.

For Anthony, it was like viewing the world permanently through a pair of sunglasses. It was only a matter of time, the doctors agreed, before the lights went out completely.

At age 6, Anthony became one of the first patients in human clinical trials for Spark Therapeutics’s experimental gene therapy to treat this rare form of congenital blindness.

Related article:  Parents of acutely ill newborns find answers through DNA sequencing

After the treatment Anthony had fresh observations about my eye color and wedding pictures that had been on our walls for years. Follow-up eye exams measuring visual acuity and light sensitivity confirmed what was plainly obvious: Gene therapy hadn’t only halted Anthony’s vision loss; it had yielded measurable vision gain. The permanent sunglasses weren’t off completely, but they were several shades brighter.

Read full, original post: Genomic Science Kept My Boys from Going Blind

Outbreak Daily Digest
Biotech Facts & Fallacies
Talking Biotech
Genetics Unzipped
Video: Test everyone – Slovakia goes its own way to control COVID

Video: Test everyone – Slovakia goes its own way to control COVID

As Europe sees record coronavirus cases and deaths, Slovakia is testing its entire adult population. WSJ's Drew Hinshaw explains how ...
mag insects image superjumbo v

Disaster interrupted: Which farming system better preserves insect populations: Organic or conventional?

A three-year run of fragmentary Armageddon-like studies had primed the journalism pumps and settled the media framing about the future ...
dead bee desolate city

Are we facing an ‘Insect Apocalypse’ caused by ‘intensive, industrial’ farming and agricultural chemicals? The media say yes; Science says ‘no’

The media call it the “Insect Apocalypse”. In the past three years, the phrase has become an accepted truth of ...
globalmethanebudget globalcarbonproject cropped x

Infographic: Cows cause climate change? Agriculture scientist says ‘belching bovines’ get too much blame

A recent interview by Caroline Stocks, a UK journalist who writes about food, agriculture and the environment, of air quality ...
organic hillside sweet corn x

Organic v conventional using GMOs: Which is the more sustainable farming?

Many consumers spend more for organic food to avoid genetically modified products in part because they believe that “industrial agriculture” ...
benjamin franklin x

Are most GMO safety studies funded by industry?

The assertion that biotech companies do the research and the government just signs off on it is false ...

Environmental Working Group: EWG challenges safety of GMOs, food pesticide residues

Known by some as the "Environmental Worrying Group," EWG lobbies for tighter GMO legislation and famously puts out annual "dirty dozen" list of fruits and ...
m hansen

Michael Hansen: Architect of Consumers Union ongoing anti-GMO campaign

Michael K. Hansen (born 1956) is thought by critics to be the prime mover behind the ongoing campaign against agricultural biotechnology at Consumer Reports. He is an ...
News on human & agricultural genetics and biotechnology delivered to your inbox.
Optional. Mail on special occasions.
Send this to a friend