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.
