Title:Gene Therapy, A Novel Therapeutic Tool for Neurological Disorders: Current Progress, Challenges and Future Prospective
Volume: 20
Issue: 3
关键词:
CRISPR / Cas9，载体，siRNA，基因编辑，神经修复，临床试验。
摘要: Neurological disorders are one of the major threat for health care system as they put enormous
socioeconomic burden. All aged populations 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
is 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 lysosomal storage disease. However, there are some limitations such as immunogenic
reactions non-specificity of viral vectors and a lack of effective biomarkers to understand the efficacy
of therapy. Considerable progress has been made to improve vector design, gene selection and
targeted delivery. This review article deals with the current status of gene therapy in neurological disorders
along with its clinical relevance, challenges and future prospective.
