Gene Therapy in Neurological Disorders

Gene therapy is a revolutionary approach to treating neurological disorders by targeting their genetic causes. This cutting-edge technique involves the introduction, alteration, or silencing of genes within a patient’s cells to correct defective genes or compensate for missing ones. For conditions like spinal muscular atrophy (SMA), gene therapy has already shown remarkable success, offering life-changing results by restoring lost motor function. Research is ongoing to explore the potential of gene therapy for treating other neurological conditions such as Parkinson’s disease, Alzheimer’s disease, and Huntington’s disease. The goal of gene therapy in neurology is to provide long-lasting, potentially curative treatments for conditions that were previously thought to be untreatable. While challenges remain, including delivery methods and safety concerns, the future of gene therapy in neurology looks promising, offering hope for patients with genetic neurological disorders.

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