The experimental drug has startling powers: It can turn down a mutant gene in a patient’s body, stopping the production of proteins that cause a terribly painful rare disease.
A crucial, late-stage clinical trial showed that the drug works — and that it’s safe. And now the biotech company behind it, Alnylam, is poised to bring this first-of-its-kind therapy to market.
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What about spherical nucleic acids? These are now in human trials for giloblastoma:
https://en.wikipedia.org/wiki/Spherical_nucleic_acid
https://news.northwestern.edu/stories/2017/may/spherical-nucleic-acid-drug-human-brain-cancer-glioblastoma/
Fascinating article about a complex subject, well covered
It’s exciting to learn about the scientific struggle to get safe RNAi drugs.
These heroes who invest in scientific progress taking risks, deserve our admiration!