AI Article Synopsis
- Gene therapy shows promise for treating retinal diseases, and the research involves creating tools for studying disease mechanisms and therapeutic testing.
- Researchers cloned the PDE6β gene and recreated a mutation found in the rd10 mouse, using a specialized plasmid vector for their experiments.
- They developed plasmids that express both normal and mutated PDE6β fused with the GFP gene to track protein expression in retinal pigment epithelial cells, advancing gene therapy for retinitis pigmentosa.
Article Abstract
Gene therapy has the potential for treating retinal diseases, and we have been developing delivery vehicles and expression vectors for this purpose. In this short communication, we describe the generation of tools for both in vitro studies of the disease mechanism and for in vivo testing of therapeutic approaches. We have cloned the PDE6β gene and also recreated the same mutation present in the rd10 mouse using an optimized plasmid vector. To allow visual detection, we have also generated, through site-directed mutagenesis, plasmids expressing the normal and mutated PDE6β gene fused with the GFP gene. Next, we have transfected retinal pigment epithelium cells with the different vectors and detected the protein expression of both the normal and mutated PDE6β. With this work we have created gene therapy tools for in vitro and in vivo studies of retinal disease-causing mutations, namely for the PDE6β, implicated in retinitis pigmentosa.
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| http://dx.doi.org/10.1016/j.jbiotec.2018.07.030 | DOI Listing |
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