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https://theprogressnetwork.substack.com/p/the-deaf-will-hear-and-the-blind>
"There are more than 100 people in the United States, blind as of a few years
ago, who can now see. I don’t mean that in a cutesy “they got a stronger
glasses prescription” kind of way. These people were born with a rare
condition, Leber congenital amaurosis (LCA), that causes total blindness by
early adulthood.
In 2017, a medicine called Luxturna, which restores the vision of LCA patients,
became the first gene therapy approved by the FDA for an inherited condition.
It works by replacing a defective gene that normally produces a protein that
the retina needs to respond to light. Just shy of a decade later, as the
husband-and-wife scientist team behind Luxturna accepted a life sciences
breakthrough prize at the “Oscars of Science” last month, nearly all eligible
LCA patients in the US have received the treatment. Luxturna isn’t a cure, but
as the prize page reports, these children can now “attend regular schools, play
outside at night, and in some cases even qualify for driver’s licenses.” The
treatment is one-and-done, and, so far, it lasts.
Luxturna isn’t the only gene therapy in the news lately. In April, the FDA also
approved the first gene therapy for genetic deafness: Otarmeni, which, like
Luxturna, targets a faulty gene. Both treatments affect small numbers of
people—Otarmeni treats a form of hearing loss that afflicts about 50 newborns
annually in the US—but they are small miracles in that they are an option at
all, as gene therapy research had slogged uphill for decades."
Cheers,
*** Xanni ***
--
mailto:xanni@xanadu.net Andrew Pam
http://xanadu.com.au/ Chief Scientist, Xanadu
https://glasswings.com.au/ Partner, Glass Wings
https://sericyb.com.au/ Manager, Serious Cybernetics
