Mutations in the RHO gene are the main cause of dominant forms of retinitis pigmentosa (RP). RHO dictates the production of rhodopsin, a transmembrane protein present in large quantities in the outer segment of rod photoreceptor cells which, together with 11-cis-retinal, initiates the phototransduction cascade. Mutations in RHO often cause photoreceptor toxicity because they negatively affect the function of the...
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In recent years, a powerful gene-editing technology called CRISPR-Cas9 has revolutionized biology turning the process of genome modification into an easy and inexpensive process. This revolutionary technique makes use of molecular tools that allow precise identification of DNA fragments –for example a gene– and either introduce modifications, delete a genomic fragment, or even insert a new DNA fragment or a...
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