Tosolini, AP;
Morris, R;
(2016)
Viral-mediated gene therapy for spinal cord injury (SCI) from a translational neuroanatomical perspective.
Neural Regeneration Research
, 11
(5)
pp. 743-744.
10.4103/1673-5374.182698.
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Abstract
Repairing spinal cord injury (SCI) is one of the most challenging endeavours currently faced by neuroscientists. One promising therapeutic avenue to reverse this once considered permanent condition is gene therapy, however progress has been hampered by the anatomical intricacy of the spinal cord itself as well as by the sheer complexity of the molecular cascades of events that take place in the injured cord. In this perspective article, we will first discuss the priority for functional regeneration. We will then review the two main therapeutic strategies to offset the deleterious outcomes of SCI and facilitate axonal regeneration, namely the reduction of the inhibitory environment around the injury site and the up-regulation of genes that are instrumental to axonal regeneration. Further, we will provide evidence that the delivery of genes with neuroregenerative properties after a SCI has the potential to offset the deleterious effects of molecules that halt regeneration after a spinal cord injury. We will subsequently consider the three most common systems currently used to shuttle therapeutic genes in various animal models of spinal cord injury, i.e., systemic, intraspinal and intramuscular delivery techniques. We will argue that the intramuscular delivery system has several advantages over the other gene delivery methods including that it is minimally invasive and does not trigger an immune response in the spinal cord. We will then reflect on some of the problems associated with the permanent transgene expression in the central nervous system and will discuss the use of transiently expressed vectors as sound alternatives for gene therapy.
| Type: | Article |
|---|---|
| Title: | Viral-mediated gene therapy for spinal cord injury (SCI) from a translational neuroanatomical perspective |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.4103/1673-5374.182698 |
| Publisher version: | http://dx.doi.org/10.4103/1673-5374.182698 |
| Language: | English |
| Additional information: | This article is available under the terms of the Creative Commons Attribution-NonCommercial-ShareAlike License (CC BY-NC-SA) (http://creativecommons.org/licenses/by-nc-sa/3.0/), which permits non-commercial use, distribution and reproduction in any medium, provided the original work is properly cited. |
| Keywords: | science & technology, life sciences & biomedicine, cell biology, neurosciences, neurosciences & neurology, central-nervous-system, motor-neuron columns, end-plates, delivery, adenovirus, expression, hindlimb |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology > Department of Neuromuscular Diseases |
| URI: | https://discovery.ucl.ac.uk/id/eprint/1521632 |
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