Gene therapy of neurodegenerative disorders

K K Jain MD (Dr. Jain is a consultant in neurology and has no relevant financial relationships to disclose.)
Originally released December 16, 1997; last updated August 30, 2020; expires August 30, 2023


This article describes gene therapy of various neurodegenerative disorders: Parkinson disease, Alzheimer disease, Huntington disease, and amyotrophic lateral sclerosis. Some of these are in clinical trials. Parkinson disease is an ideal candidate. The introduction of functional genes into the brain of patients with Parkinson disease may, for example, prove useful for replacing a defective gene, introduce a potentially neuroprotective or neurorestorative protein, or permit the physiological delivery of a deficient neurotransmitter. Potential gene therapy approaches to Alzheimer disease are based on neurotrophic factors and not neurotransmitters. The optimal method of gene therapy is ex vivo involving implantation of genetically engineered cells secreting nerve growth factor. A clinical trial for amyotrophic lateral sclerosis was conducted with implantation of genetically modified cells.

Historical note and terminology

Gene therapy can be broadly defined as the transfer of defined genetic material to specific target cells of a patient for the ultimate purpose of preventing or altering a disease. Carriers or delivery vehicles for therapeutic genetic material are called vectors, which are usually viral, but several nonviral techniques are being used as well. Gene therapy usually implies the introduction of altered genes into the body of a patient instead of just the products of cells with altered genes. The term "genetic engineering" applies to the genetic manipulation of living cells, as well as implantation of genetically engineered cells into the living body, which is a form of gene therapy. Neurosurgeons refer to gene therapy as "cellular and molecular" neurosurgery. The history and basics of gene therapy are described in the article on introduction to gene therapy.

Gene therapy of Parkinson disease, Alzheimer disease, Huntington disease, and amyotrophic lateral sclerosis will be described in this article. RNA interference (RNAi)-based techniques, which can be employed to reduce expression of specific genes, hold great promise as therapy for these types of neurodegenerative disorders.

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