Gene therapy is one 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.
Keywords: Gene therapy, Neurodegenerative disorder, Neuropathy, CNS, AAV, Vectors.Gene therapy, Neurodegenerative disorder, Neuropathy, CNS, AAV, Vectors.
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