Shoot the messenger: RNA editing is here
— RNA molecules with catalytic activities — for RNA-guided trans-splicing of targeted RNA sequences in vitro and in. “These catalytic RNAs don’t come from mammalian systems,” says Sullenger, who is at Duke University. “In evolution, a lot of the [catalytic] functions have been replicated by proteins.
Seong-Wook Lee, a former postdoctoral researcher in Sullenger’s lab, continued the work in his native South Korea, and Rznomics has emerged to take it into the clinic but with the emphasis switched to cancer. Rznomics’ lead program, RZ-001, for which it has received FDA clearance to conduct a US clinical trial for liver cancer, is designed to sensitize to ganciclovir cancer cells that overexpress human telomerase reverse transcriptase.
Unlike ADAR-based editing, which is limited to single-base A-to-I substitutions, both Ascidian’s and Rznomics’ approaches enable large-scale changes to be introduced in a mature mRNA transcript. “ADAR is like fixing typos. The splicing approaches are rewriting paragraphs,” says Sullenger.
Even if ADAR-based editing firms are limited to making single-base changes, the resulting biological consequences can be large. Modulating the levels of a transcription factor by increasing its half-life by even 10% can have a significant effect, Aiyar says. Introducing a dominant negative mutation to a protein can also have an outsized effect, Platenburg says. With the notable exception of AATD-directed therapies, most ADAR-based programs remain under wraps for now.
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