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8
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1 scientist
9
Evidence
6
Balance
9
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VKC
Vignesh Kartik Chundru
Reviewed on 20 May 2025
9
Evidence
6
Balance
9
Clarity

I would argue it's not the "first", but this is a very exciting advance in gene therapies for rare disease.

The article does a good job of explaining why the medical community is so excited about this, but it doesn't touch on the limitations. This is not possible for all disorders, in particular those affecting prenatal development

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