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

Introduction

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.

Challenges

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

Objectives

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:

1. Simplify the chemistry and the compounds to such an extent that a SNAP-ADAR kit is obtained.
2. Enable Life Scientists to perform the required chemistry without special equipment or chemistry skills.
3. Include a reliable and simple protocol to perform efficient and precise editing reactions in mammalian primary cells.

Commercial Potential

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, such as signaling cues and metabolic pathways.
• Target screening and target validation in drug discovery efforts, which are currently ongoing in the field of RNA base editing.

Future Steps

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.