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Myotic Dystrophy Treatment Breakthrough
PLUS: Mysteries Surround Ehlers-Danlos Syndrome
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Hi friends 👋🏼,
From cancer to blindness - gene therapy is revolutionizing medicine. We’ve got four stories for you this week, taking three of your requests:
Myotic Dystrophy Treatment Breakthrough READER REQUEST
Mysteries Surround Ehlers-Danlos Syndrome READER REQUEST
Measles to Kill Medulloblastoma READER REQUEST
Let There Be Light: Gene Therapy for Blindness
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✋🏼🤚🏼 Myotonic Dystrophy Treatment Breakthrough
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Congenital Myotonic Dystrophy (CMD) is an inherited genetic disorder characterized by progressive muscle wasting and weakness, among other symptoms. For example, imagine opening a door and having difficulty releasing your grip on the doorknob.
1/ CMD is caused by a mutated DMPK gene. Specifically, sections of the DMPK gene are incorrectly repeated thousands of times. As a result, the abnormally large gene produces an unusually large mRNA, producing a nonfunctional protein that causes the disease.
2/ Avidity Biosciences has developed AOC 1001, designed to address CMD's root cause.
Here’s how AOC 1001 works👇🏼
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