What is gene therapy?

Gene therapy has shown promising results in the treatment of hereditary ocular diseases such as retinitis pigmentosa and Usher syndrome. These diseases are caused by specific genetic mutations that affect the structure or function of photoreceptors in the retina. Gene therapy can provide healthy copies of the defective gene or introduce genes that promote the survival and function of photoreceptors, potentially halting or slowing the disease's progression and improving vision in patients.

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