Harnessing CRISPR-Cas9 Gene Editing for the Eradication of Inherited Retinal Diseases in Purebred Dogs: A Path to Preservation and Health
Harnessing CRISPR-Cas9 Gene Editing for the Eradication of Inherited Retinal Diseases in Purebred Dogs: A Path to Preservation and Health
Ali Sabeeh Ali1, Noor Abdulaala Kadhim2, Entissar Mansour Abdul Rasool3, M. Al-Erjan4, Qais R. Lahhob5*, Mustafa Mudhafar6,7
ABSTRACT
The publication looked at CRISPR-Cas9 gene editing as one of the potential therapeutic strategies for LCA2 in pure bred dogs. The gRNA was designed in such a way that it would bind the canine RPE65 gene associated with LCA2. Also, an LV was designed to deliver the Cas9 nuclease and the gRNA into the target cells. The vector was further confirmed in immortalized canine retinal pigment epithelial (RPE) cells, therefore confirming modification to the target sequence. The research involved 8 pure bred dogs that had been diagnosed with LCA2 and who possesses the intended mutation. OCT and ERG were done at baseline and then at 4 weeks, 8 weeks and 12 weeks after the injection to analyse the changes in the retinal thickness and function. All the results highlighted long-standing and Statistically significant increases in retinal thickness in the treatment group compared to the control group at weeks 4, 8 and 12 after injection. The same is true with the treated group which exhibited marked increases in the a-wave and the b-wave in the electroretinogram (ERG), indicating better retinal function compared to the other group. These improvements were most apparent in the treated group, at the twelfth week after injection. The study shows that with the help of CRISPR-Cas9 gene editing, structural and functional changes occur in the canine retina in case of LCA2. In their study, the authors have provided strong grounds for considering this therapy beneficial in the hereditary retinal diseases in purebred dogs under the condition that further research will be conducted. More research endeavour is needed in order to evaluate the residual effects, mode of delivery, efficacy, and the issues of safety and ethicality of an innovation. Further research is therefore needed in order to assess the potential longer-term outcomes, implementation strategies, effectiveness and the issues of safety translation.
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