RNA Trans-splicing
The Splice is Right
RNA carries the genetic instructions to make protein. Before the protein can be made, certain unnecessary portions of the RNA are removed. RNA trans-splicing is a technology that hijacks this naturally occurring phenomenon in order to remove the mutated sections of the MECP2 protein RNA and replace it with healthy versions.
RNA trans-splicing is attractive for two main reasons. First, a single RNA trans-splicing therapeutic could treat 97% of all Rett patients. An additional therapeutic would be created to address the remaining 3%. Second, it avoids any possibility of producing too much of the MECP2 protein.
Until recently RNA trans-splicing has been a rather obscure genetic medicine approach without much progress. Academics and biotech companies more recently have embraced this approach.
We’ve funded Stuart Cobb at the University of Edinburgh for this approach and are seeking additional opportunities to advance RNA trans-splicing.
"Like other parents of children with Rett syndrome, Coenraads wishes scientists would hurry up and develop treatments. But as the founder and CEO of the Rett Syndrome Research Trust, she’s in a position to do something about it.”
RSRT featured in a recent Science Magazine article: Buoyed by 'milestone' clinical result, RNA editing is poised to treat diseases.
