Regulated gene expression for Huntington's disease therapy

Regulated gene expression for Huntington's disease therapy

Huntington's Disease (HD) is chronic neurodegenerative disease. HD is caused by a defect in the Huntingtin gene. As a consequence of this defect, mutated proteins are produced in the brain. These mutated proteins accumulate in the brain and destroy the neurons, causing loss of their functions. So far, the treatment of the disease is only palliative. Gene therapy is a technique for correcting defective or missing genes that otherwise cause a disease. This can be done by inserting a functional copy of the gene into the appropriate cells of the patient, to restore body function.