Gene therapy to restore vision in mice

Blindness is a global problem where a person cannot see anything, including light. Researchers find novel ways like gene therapy to impart vision, and they initially experimented with mice. They claimed to restore vision in totally blind mice with the help of gene therapy.

Physiology of vision process

Opsins are a group of proteins, that is light sensitive through the presence of chromophore retinal found in the photoreceptor cells of the retina. These opsins only covalently bind to vitamin-A based retinaldehyde in both rods and cones. This starts a cascade of signals when the light activates the photoreceptor cells in the retina. This causes pulsations, which propagates by creating an impulse through the optic nerve to the brain.

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Several disease conditions like macula degeneration, retinitis pigmentosa were causing damage to the photoreceptor cells, which in turn leading to blindness.

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The study

Scientists said that they can still restore vision even if the photoreceptor cells are impaired. This is by a group of neurons, which we say bipolar cells, can help to evade blindness. When photoreceptor cells failed, other neuronal cells would remain intact.

This is possible when we insert the MCO1 opsin gene to the intact bipolar cells in the retina, which is downstream to the photoreceptors. Hence, the ability to see objects or light enhances.

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This gene therapy could overcome other treatments such as replacement of opsin, invasive surgery, and all that. Also, this treatment is effective in causing 20/60 vision in patients. Clinical trials are necessary to depict a clear picture of showing how it works when it comes to fast-moving objects, and whether it affects vision quality.

Experiments with mice did not yield any harmful effects upon the administration of genes. If everything goes well, they can successfully implement the treatment, and it will definitely be an alternative to the retinal prosthesis such as retinitis pigmentosa.

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Source: Medical Xpress

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