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Personalized CRISPR therapies could soon reach thousands — here’s how

Urnov and Kassim propose regulatory frameworks to help disseminate personalized CRISPR treatments to rare disease patients who need them. Without better regulations, every guide RNA nucleotide change necessitates a long (~4 year) process to obtain approval. Many rare diseases can progress quickly, so this time period is often deadly for patients.


A fresh approach to trialling treatments for rare genetic diseases could make their production an economically viable prospect at last.

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