Laser-assisted targeted gene delivery to degenerated retina improves retinal function |
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Authors: | Subrata Batabyal Sanghoon Kim Weldon Wright Samarendra Mohanty |
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Affiliation: | Nanoscope Technologies LLC, Bedford, Texas, USA |
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Abstract: | Delivery of therapeutic genes into retina is proving to reverse degeneration and restore vision, however, viral vector-based gene delivery is prone to immunorejection, inflammatory/immune-response and nontargeted. Here, we report nonviral gene delivery and expression of opsin encoding genes in mouse retina in-vitro and in-vivo by use of pulsed femtosecond laser microbeam. In-vitro patch-clamp recording of the opsin-sensitized retinal cells and visually evoked in-vivo electrical recording from laser-transfected eye of mouse with degenerated retina showed functional response. The ultrafast laser-based naked gene delivery showed minimal damage and reliable expression of therapeutic opsin in cell membrane of the selected cells and in targeted retinal region. Laser-based “naked DNA gene therapy” in a spatially targeted manner will pave the way for treatment of inherited retinal diseases.![image](/cms/asset/36480fa3-ea0c-49c2-8117-13cf9d697605/jbio202000234-gra-0001.png) |
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Keywords: | gene therapy optical gene delivery optogenetics retinal dystrophy |
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