RNA, a short-lived cousin to its better-known partner DNA, is the blueprint for protein production in cells. [Biotech entrepreneur Joshua] Rosenthal told the Atlas investors about how squid and octopuses make prolific use of an enzyme called ADAR to catalyze thousands of single-letter changes to their RNA code. Those minor edits alter the structure and activity of proteins that control electrical impulses in the animals’ nerves.
Humans have ADAR enzymes in our bodies, too, where they do the same thing.
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Unlike DNA editing, which is permanent, the effects of RNA editing are reversible, since cells are constantly churning out new copies of RNA. If Rosenthal’s RNA editors work in humans, they could be used to repeatedly treat genetic diseases without confronting the unknown, long-term risks of permanent DNA editing with CRISPR. More importantly, they would offer a new strategy for treating conditions like pain or inflammation, in which a patient needs just a temporary fix. RNA editing could also be easier to turn into a therapy than CRISPR. Since ADAR already exists in our cells, in theory all that’s needed is a guide RNA to lasso the enzyme and tell it where to go.
Read full, original post: Watch out, CRISPR. The RNA editing race is on
