RNA-targeted therapy in Stargardt disease

Dr Cerys Manning - University of Manchester - £24,920

This research project explores a promising new approach to treating Stargardt disease, a genetic condition that causes progressive vision loss. By targeting faulty messages within cells, the research aims to preserve sight and slow disease progression.

What is the problem?

Stargardt disease develops due to mutations in the ABCA4 gene. The severity and age of onset depend on the specific mutations a person carries, some allow partial function, while others cause more severe damage. Currently, there is no cure, and affected individuals face progressive vision loss.

What are they doing?

The research team are using CasRx, a molecular tool that acts like “scissors” to selectively cut harmful ABCA4 mRNA messages before they produce faulty proteins. mRNA's primary role is to act as a messenger, carrying the instructions for making proteins from the DNA to where proteins are made in the cell. By removing these harmful messages, the proportion of functional protein can be increased, potentially slowing or halting disease progression.

CasRx is flexible and can target multiple types of mutations, making it a versatile approach for Stargardt disease.

How can this help?

This approach is designed to be safer than permanent genome editing and could be translated into clinical treatments more rapidly. If successful, it could offer a practical way to slow or prevent vision loss in people living with Stargardt disease.