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Site-directed RNA Editing by Engineered Human ADAR2

Efficient system to selectively re-code genetic information within mRNAs with potential to restore structure/function of defective proteins

Background

Unlike DNA editing tools, which cause permanent alterations to the genetic sequence, RNA editing offers reversible changes in gene expression, eliminating the risk of lifelong off-target genetic modifications.

Adenosine deaminases that act on RNA (ADARs) are responsible for the natural and evolutionarily conserved mechanism by which adenosines are converted to inosines. During translation, inosine is read as guanosine, therefore codons in mRNAs are recorded. However, the natural targeting of ADARs relies on double-stranded RNA binding domains that recognize secondary and tertiary structures within the surrounding RNA. Such specific and often complex higher order RNA structures would not serve us to guide RNA editing.

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