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

✋🏼🤚🏼 Myotonic Dystrophy Treatment Breakthrough

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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