Gene therapy, a novel therapeutic tool for neurological disorders: Current progress, challenges and future prospective.

Gene therapy, a novel therapeutic tool for neurological disorders: Current progress, challenges and future prospective. Curr Gene Ther. 2020 Jul 15;: Authors: Iqubal A, Iqubal MK, Khan A, Ali J, Baboota S, Haque SE Abstract Neurological disorders are one of the major threat for health care system as it puts enormous socioeconomic burden. All aged population are susceptible to one or other neurological problems with symptoms of neuroinflammation, neurodegeneration and cognitive dysfunction. At present available pharmacotherapeutics are insufficient to treat these diseased conditions and in most cases they provide only palliative effect. It was also found that the molecular etiology of neurological disorders are directly linked with the alteration in genetic makeup which can be inherited or triggered by the injury, environmental toxins and by some existing disease. Therefore, to take care of this situation, gene therapy has emerged as an advanced modality that claims to permanently cure the disease by deletion, silencing or edition of faulty genes and by insertion of healthier genes. In this modality, vectors (viral and non-viral) are used to deliver targeted gene into a specific region of the brain via various routes. At present, gene therapy has shown positive outcomes in complex neurological disorders, such as Parkinson's disease, Alzheimer's disease, Huntington disease, Multiple sclerosis, Amyotrophic lateral sclerosis and in lysos...
Source: Current Gene Therapy - Category: Genetics & Stem Cells Authors: Tags: Curr Gene Ther Source Type: research