Unsplash/Warren Umoh
29 March 2023
Chinese researchers have restored vision in a mouse model of retinitis pigmentosa (RP) using a new form of CRISPR gene editing.
The research, which was published in Journal of Experimental Medicine, outlines how the gene editing system can be programmed to correct many different types of genetic mutation – irrespective of the location on the genome.
Scientists highlight that once the mutation was corrected in the mice with RP, the progressive cell loss was reversed and there was a “substantial rescue” of photoreceptors.
“The treated mice exhibited significant responses in electroretinogram and displayed good performance in both passive and active avoidance tests. Moreover, they presented an apparent improvement in visual stimuli-driven optomotor responses and efficiently completed visually guided water-maze tasks,” the authors highlighted.
Professor Kai Yao, of Wuhan University of Science and Tech, emphasised that more work needs to be done to establish the safety and efficacy of the genome editing system in humans.
“However, our study provides substantial evidence for the in vivo applicability of this new genome-editing strategy and its potential in diverse research and therapeutic contexts, in particular for inherited retinal diseases such as retinitis pigmentosa,” Yao said.
The research, which was published in Journal of Experimental Medicine, outlines how the gene editing system can be programmed to correct many different types of genetic mutation – irrespective of the location on the genome.
Scientists highlight that once the mutation was corrected in the mice with RP, the progressive cell loss was reversed and there was a “substantial rescue” of photoreceptors.
“The treated mice exhibited significant responses in electroretinogram and displayed good performance in both passive and active avoidance tests. Moreover, they presented an apparent improvement in visual stimuli-driven optomotor responses and efficiently completed visually guided water-maze tasks,” the authors highlighted.
Professor Kai Yao, of Wuhan University of Science and Tech, emphasised that more work needs to be done to establish the safety and efficacy of the genome editing system in humans.
“However, our study provides substantial evidence for the in vivo applicability of this new genome-editing strategy and its potential in diverse research and therapeutic contexts, in particular for inherited retinal diseases such as retinitis pigmentosa,” Yao said.
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