Gene Therapy for the Treatment of Neurological Disorders: Amyotrophic Lateral Sclerosis

Gene therapy is a powerful tool for treating diseases, including neurological disorder such at amyotrophic lateral sclerosis. When delivered to the CNS, gene therapy vectors can provide prosurvival signals to neurons, knock down the expression of toxic proteins, or restore lost function. How to best deliver this type of therapeutic depends on the nature of the disease and the expected function of the transgene. Here we describe a method for parenchymal injection into rodent models, allowing for localized delivery of gene therapy vectors and other therapeutic molecules. This technique has been a robust mechanism for proof-of-principle experiments.