Title:Exploring the Role of Gene Therapy for Neurological Disorders
Volume: 21
Issue: 1
关键词:
基因治疗，神经退行性疾病，神经病变，中枢神经系统，AAV，载体
摘要: Gene therapy is one of the frontier fields of medical breakthroughs that poses as an effective
solution to previously incurable diseases. The delivery of the corrective genetic material or a
therapeutic gene into the cell restores the missing gene function and cures a plethora of diseases, incurable
by the conventional medical approaches. This discovery holds the potential to treat many
neurodegenerative disorders such as muscular atrophy, multiple sclerosis, Parkinson’s disease (PD)
and Alzheimer’s disease (AD), among others. Gene therapy proves as a humane, cost-effective alternative
to the exhaustive often arduous and timely impossible process of finding matched donors
and extensive surgery. It also overcomes the shortcoming of conventional methods to cross the
blood-brain barrier. However, the use of gene therapy is only possible after procuring the in-depth
knowledge of the immuno-pathogenesis and molecular mechanism of the disease. The process of
gene therapy can be broadly categorized into three main steps: elucidating the target gene, culling
the appropriate vector, and determining the best mode of transfer; each step mandating pervasive research.
This review aims to dissertate and summarize the role, various vectors and methods of delivery
employed in gene therapy with special emphasis on therapy directed at the central nervous
system (CNS) associated with neurodegenerative diseases.
