CRISPR has delivered on a key aspect of its promise as a platform technology: going from disease 1 to disease 2 requires changing only 20 nt in an RNA.
This is exactly what @intelliatx just did: the HAE data look as good as the TTR ones.
YAY+BRAVO! statnews.com/2022/09/16/int…
Teacher, Clinical Supervisor
Clinical Associate Professor, Psychiatry
University of Florida
Affiliation is for identification only. All content and opinions are my own.
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