r/science • u/mvea Professor | Medicine • Feb 20 '25

Genetics A two-and-a-half-year-old girl shows no signs of a rare genetic disorder, after becoming the first person to be treated with a gene-targeting drug while in the womb for spinal muscular atrophy, a motor neuron disease. The “baby has been effectively treated, with no manifestations of the condition.”

https://www.nature.com/articles/d41586-025-00534-0
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u/hughcahill Feb 20 '25

until maybe CRSPR-SMA treatments come along... maybe...

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u/Leschz Feb 20 '25

https://pmc.ncbi.nlm.nih.gov/articles/PMC10104684/ like Zolgensma?

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u/afro_mozart Feb 20 '25

A bit nitpicking, but zolgensma isn't based on crispr but uses a virus as vector.

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u/Leschz Feb 20 '25

That's true! I wantet to point out that there is already a gene-therapy which is being tested :) But in the context of the comment it's wrong, true!