<
https://www.science.org/content/article/buoyed-milestone-clinical-result-rna-editing-poised-treat-diseases>
"Monica Coenraads admits that as a first-time parent who hadn’t spent much time
around children, she was slow to notice that something was wrong with her
daughter, Chelsea. By the time Chelsea was 1 year old, however, her development
had obviously stalled and even begun to reverse. She only learned to speak one
word and soon stopped saying anything. Chelsea could only walk if someone held
her upright. She lost the ability to grasp and instead began “making repetitive
movements with her hands” such as clapping, says Coenraads, whose family lived
in Virginia at the time.
“I was desperate for a diagnosis,” Coenraads says. But when she finally got
one, when Chelsea was 2, “it was a double gut punch.” Not only did Chelsea have
Rett syndrome, an untreatable neurological disorder, but scientists knew little
about the condition. They understood it primarily affected girls and was likely
due to a mutation, but they hadn’t identified the genetic culprit.
That was in 1998. Today, Coenraads and her husband are still caring for
Chelsea, now 28, who is unable to speak, walk, or use her hands. She requires
medications to quell seizures, reduce anxiety, and help her sleep. The genetic
glitches behind the disease are now known: usually mutations in the gene
MECP2, which controls gene activity in many organs, including the brain. Like
other parents of children with Rett syndrome, Coenraads
wishes scientists
would hurry up and develop treatments. But as the founder and CEO of the Rett
Syndrome Research Trust, she’s in a position to do something about it.
In addition to funding a range of strategies to correct or replace faulty DNA,
her organization is backing an unconventional approach. The trust has poured
$8.5 million—more than 10% of the money it has doled out for research—into
efforts to edit the strands of RNA encoded by mutated
MECP2. In doing so they
are bolstering a nascent but promising approach to treating diseases: editing
the RNA blueprints for proteins. “Our goal is to fertilize the field,”
Coenraads says."
Via
Fix the News:
https://fixthenews.com/276-regenerative-city/
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
