Gene Therapy for Neurodegenerative Diseases

Abstract

In today’s world, genes are very useful components for the treatment of many diseases. Gene therapy can be considered as the insertion of a gene to the cell in order to correct a cellular function or to treat a disease. Vectors like viral and non-viral vectors are used in gene therapy as carriers. It has the potential to deliver therapeutic advantage to lot of people with neurodegenerative diseases by several means, counting direct improvement of pathogenic mechanisms, neuroprotection, neuro-restoration, and symptoms control. Therapeutic efficacy is therefore reliant on knowledge of the disease pathogenesis and the required temporal and spatial specificity of gene expression. An additional critical challenge is achieving the most complete transduction of the target structure while avoiding leakage into neighboring regions or perivascular spaces. The gene therapy field has recently entered a new technological era, in which interventional MRI-guided convection-enhanced delivery (iMRI-CED) is the gold standard for verifying accurate vector delivery in real time. Gene therapy can be promising treatment goal for many neurodegenerative diseases like Alzheimer’s disease, Parkinson’s disease, multiple sclerosis and epilepsy. With the help of vectors like viral and non-viral vectors, Gene therapy can bring treatment of these diseases to reality. The availability of this advanced neurosurgical technique may accelerate the translation of the promising preclinical therapeutics under development for neurodegenerative disorders, including Parkinson's, Huntington's, and Alzheimer's diseases.