Retina UK awards new research funding

Prof Lako’s new project will build on earlier work funded by Retina UK, in which she and her team investigated how defects in the “spliceosome”, an important and complex structure within cells, contribute to retinal degeneration in retinitis pigmentosa (RP). Working with collaborators across four centres in the UK and Germany, Prof Lako will now use cell-based disease models to explore a gene therapy approach to addressing these defects, focusing on a spliceosome gene called PRPF31.

This study will provide a unique opportunity for rapid proof-of-concept for the therapy, which could ultimately provide the basis for translation into a Phase I/II clinical trial for people with RP caused by mutations in PRPF31. Between them, faulty spliceosome genes are a relatively major cause of RP, so the outcomes of this project should also be applicable to the development of treatments for a wider proportion of our community.

Prof Lako told us: “I am delighted to receive this award and to move our work to the translational stage. I am very grateful to Retina UK’s supporters for making this progress possible”.

Find out more on the project and the function of the spliceosome.