Site-directed RNA editing

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site-directed-editing

A schematic of our system for site-directed RNA editing.

Imagine if we could direct RNA editing to where we wanted it to go. If we could then genetic mutations could be erased or protein function retuned in ingenious ways. For example, if you were unlucky enough to be born with the W1282X premature termination codon in your gene for the Cystic Fibrosis Transmembrane Conductance Regulator, then you could edit the codon back to tryptophan in mRNAs. Or if you were suffering from chronic pain, maybe you could subtly shift the voltage-sensitivity of the right Na channel. The trick would be to redirect the activity of the RNA editing enzyme ADAR. We’ve figured out an approach to doing just that. By coupling ADAR’s catalytic domain with an antisense RNA oligo, we can direct it to specific addresses along the RNA registry. To see how, check out these publications by Maria Fernanda Montiel-González and Isabel C. Vallecillo-Viejo:

An efficient system for selectively altering genetic information within mRNAs

Correction of mutations within the cystic fibrosis transmembrane conductance regulator by site-directed RNA editing