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RNA Editing

Amount Funded: $7.4 Million

Hijacking Nature

DNA is the master blueprint for life, containing all the instructions needed to build and maintain an organism. DNA lives in the nucleus of cells where it is kept safe and sound. When the cell needs to use some of the DNA instructions to make proteins, it creates a temporary copy called RNA which leaves the nucleus and goes to the ribosomes, where proteins are made.

Typos can sometimes happen when DNA is being copied into mRNA. The body uses a naturally occurring enzyme called ADAR that acts like a spell checker that locates and corrects typos. Scientists have figured out how to hijack ADAR to correct specific mutations

Gail Mandel, PhD

Oregon Health & Science University

With over a decade of RSRT funding, Gail Mandel, PhD, and post-doctoral fellow, John Sinnamon, PhD, were the first to pursue RNA editing for Rett. The Mandel lab successfully corrected a sufficient amount of RNA in mice to improve symptoms. This encouraging progress by the Mandel lab catalyzed the launch of Vico Therapeutics and put RNA editing on the map as a viable genetic medicine approach for Rett syndrome.


UC Davis

Peter Beal, PhD

Peter Beal, PhD, is a world expert in RNA editing and understanding of ADAR biology. Rather than introducing ADAR into cells, his approach recruits ADAR that is already in the cell. This approach will require dosing an individual with Rett on a regular basis. The advantage of multiple dosing is that it allows for timing and titration of the dose to be tailored to each patient in a precise way.



RSRT and ProQR, a biotechnology company in The Netherlands, are collaborating to develop small RNAs as therapeutics for Rett syndrome. Because of their small size, these RNAs can be injected and taken up like small molecule drugs and require no viral or non-viral delivery assistance. Dosing would likely be necessary several times a year with intrathecal delivery.

Shape Therapeutics


Shape Therapeutics, a biotech company based in Seattle, has developed a proprietary RNAfix technology that can precisely target a point or nonsense mutation. In 2020, they announced a Rett syndrome program. RSRT contributed to this decision by providing a number of resources, including access to our biorepository of patient cell lines.



Vico Therapeutics, a biotech based in The Netherlands, is focused on treating rare neurological disorders with antisense oligonucleotides. Vico’s strategy is to edit RNA using antisense oligonucleotide.

The scientific founder of Vico is Gail Mandel who pioneered RNA editing for Rett syndrome with substantial funding from RSRT. We also provided Vico with ccess to our biorepository of Rett cell lines to enable this research.