21 April 2021
Scientists from University College London (UCL) will investigate whether inherited retinal faults could be treated using gene editing technology.
The research, which is funded by Fight for Sight, involves using CRISPR-CAS9 base editing technology to directly repair faulty genes.
The technique will be applied to retinal organoids grown in the lab using a system developed by the researchers that splits the base editing machinery into two separate viral vectors.
The machinery recombines when introduced to the light-sensing photoreceptors within the mini retinas.
UCL research lead, Dr Jacqui van der Spuy, highlighted that the base editing technology has been adapted to overcome limitations which have so far prevented the technique from being used to treat genetic disease.
“Our study will significantly advance the application of direct base editing as a therapeutic approach for inherited retinal diseases and other genetic conditions and will accelerate base editing technologies towards a clinical treatment for patients with genetic eye conditions,” she explained.
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