Combination gene therapy to correct faulty nerves
You might be forgiven for thinking this image is of a worm but it's actually a magnified optic nerve. Nerves (neurons) are long cells and they have a sophisticated transport mechanism to move structures and biochemicals along their length. In neurodegenerative disorders, such as glaucoma and Alzheimer’s disease, this transport mechanism is faulty. So, when the cell is damaged the neuron dies because the repair process is too slow. This causes the symptoms of the disorder. As a treatment, scientists are currently developing a promising combination gene therapy where two faulty genes are replaced. This will correct the transport mechanism allowing the neurons to be repaired. The image shows how the effectiveness of the treatment is measured in the lab. The green substance on the left is moving along an optic nerve. The dots, lines and blocks were added to act like a ruler to measure how far the substance moved.
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