Simplifying the SNAP-ADAR Tool for Broad Usage in Life Science Research and Drug Discovery

RNA base editing is a promising approach to manipulate genetic information in a transient and dosable way, which breaks new ground in basic Life Science research and drug discovery.

In a preceding ERC CoG action, we have engineered the SNAP-ADAR tool, which allows for the particularly precise, efficient and programmable adenosine-to-inosine RNA base editing.

However, the broad usage of this tool is somewhat hampered by the need for designing the chemically modified guideRNAs, which currently includes some peptide bond-forming chemistry and nucleic acid gel purification.

In this PoC action, we aim to simplify the tool to make it available for a larger section of the Life Science community.

Specifically, we aim to simplify the chemistry and the compounds to such an extent that a SNAP-ADAR kit is obtained, which enables the Life Scientist to perform the required chemistry without special equipment or chemistry skills.

Furthermore, the kit would include a reliable and simple protocol to perform efficient and precise editing reactions in mammalian primary cells. Profit could particularly be generated from the selling of the chemically stabilized guideRNAs.

We envision that the product would find application in the modulation of protein function like signaling cues and metabolic pathways and may foster the target screening and target validation in drug discovery efforts, which are currently ongoing in the field of RNA base editing.

The action also contains an analysis of the IP landscape and - at the later stage of the action - we aim to involve industrial partners for commercialization.