Progresses in Gene Therapy of Neurodegenerative Disorders
Journal Title: Online Journal of Neurology and Brain Disorders - Year 2018, Vol 1, Issue 1
Abstract
Gene therapy is a technique that uses genes to treat or prevent diseases. Gene therapy to the brain faces other difficulties in addition to the general issues one faces with gene therapy. A major limiting factor for the delivery of gene to the brain is the blood brain barrier (BBB), which most gene expression vectors can not cross naturally. However, recent advances in gene transfer technology have led to promises for delivery of therapeutic genes to brain. In this small review we shall make a discussion on the recent developments of gene therapies for some important neurodegenerative disorders. Gene therapy is the administration of genetic material into the somatic cells of a patient that exerts therapeutic effects by correcting genetic defects, over expression of desired proteins or inhibition of the expression of unwanted harmful proteins [1]. For primary neurodegenerative disorders patient's condition progressively deteriorated and the current therapies remain limited to symptomatic treatments [2]. The presence of blood-brain barrier (BBB) prevents the delivery of therapeutic agents to the brain [3]. However, recent advances in gene transfer technologies show promise for delivery of therapeutic genes to brain for the replacement of defective proteins, neuroprotection, restoration of neuronal functions etc [2]. With the advent in the understanding the molecular pathologies, it is anticipated that the therapeutic genes have the ability of revolutionize the management of human diseases [4].
Authors and Affiliations
Ashim Kumar Basak, Tridip Chatterjee
Keywords
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- EP ID EP590686
- DOI 10.32474/OJNBD.2018.01.000103
- Views 79
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