Clinical trial on the horizon for RP genetic therapy

Researchers hope to use advanced gene editing technology to reverse the course of retinitis pigmentosa

09 May 2017 by Selina Powell

American researchers have used the gene-editing tool CRISPR/Cas9 to reverse the course of retinitis pigmentosa (RP) and restore vision in mice.

The study, published in Cell Research, involved using the genetic tool to prevent the degeneration of the light-sensing cells.

CRISPR/Cas9 allows scientists to target specific sections of genetic code and edit DNA at precise locations.

By deactivating certain genes, researchers were able to transform rod photoreceptors into cone photoreceptors – cells that are less vulnerable to the genetic mutations that cause RP.

Senior author, Dr Kang Zhang, explained to OT that traditional RP gene therapy approaches targeted a single gene or mutation.

However, he emphasised that since there were many mutations and genes causing RP, many patients were left without treatment options.

“Our gene therapy approach will create a universal, cost-effective gene therapy treatment for a majority, if not all, RP patients,” Dr Zhang elaborated.

Dr Zhang emphasised that a common cold virus was being used as a gene therapy vector and had a good safety profile in clinical trials.

It was expected that a preclinical study would be conducted over the coming months, and a human trial would be undertaken early next year.


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