Project Details
Description
We have pre-clinical proof-of-concept for a novel and non-obvious use of a type of gene therapy called spliceosome-mediated RNA trans-splicing (SMaRT) to treat a set of rare neurological diseases called the spinocerebellar ataxias. This project seeks funds for IP protection as well as funds to undertake some additional new analysis that is required to make our IP an attractive licensing opportunity.
Layman's description
We have developed a new type of gene therapy called RNA trans-splicing to treat a set of rare neurological diseases called the spinocerebellar ataxias. This project seeks to undertake some complex analysis that is required to complete the proof-of-concept phase.
| Status | Finished |
|---|---|
| Effective start/end date | 20/02/24 → 31/07/24 |
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Research output
- 2 Poster
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Spliceosome‐mediated RNA trans-splicing prevents the pathogenic phosphorylation of ataxin-1: ICAR 2024
Buijsen, R., Schoen, L., Jones, L., Avale, E., Petrakis, S., Gallo, J.-M. & Anthony, K., 12 Nov 2024.Research output: Contribution to Conference › Poster › peer-review
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Spliceosome‐mediated RNA trans-splicing prevents the pathogenic phosphorylation of ataxin-1
Buijsen, R., Schoen, L., Jones, L., Avale, E., Petrakis, S., Gallo, J.-M. & Anthony, K., 6 Oct 2024.Research output: Contribution to Conference › Poster › peer-review