New RP2 gene therapy for RP

Scientists have developed a new gene therapy approach that could treat retinitis pigmentosa (RP).


Genetics, virology and ophthalmology researchers from Trinity College Dublin and University College London (UCL) teamed up to deliver a normal, functioning copy of the RP2 gene, which provides instructions for making a protein essential for normal vision, into ‘mini retinas’, engineered from stem cells and containing a defective form of the gene.


The mini retinas successfully took up the functioning RP2 gene and produced the essential protein resulting in significant improvement, said PhD researcher Ciara Shortall, co-author of the study published in Stem Cell Reports.


“It is an important development that we can now reproduce so many elements of inherited disease using these mini retinas. It makes it possible for us to study in detail why people go blind and try to find ways to prevent blindness,” said Professor Michael Cheetham from UCL which led the development of the mini retinas to road-test the gene therapy.


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