Donate for RNA-Based Therapy
- CTNNB1 Syndrome is an autosomal dominant condition. While one gene is mutated, the other one is healthy.
- The RNA therapies would enhance the wild type β-catenin from the healthy CTNNB1 allele.
- Inhibition of β-catenin degradation is currently explored by:
a) Skipping exon3 that encodes the regulatory region
b) Editing of β-catenin mRNA to mutate key phosphorylation residues that govern degradation
c) Downregulation of proteins involved in the β-catenin destruction complex
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