Gene therapy for sensory neuropathy treatment

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Expert Opin Biol Ther. 2006 May;6(5):499-507.

Gene therapy for the treatment of sensory neuropathy.

Mata M, Chattopadhyay M, Fink DJ.

Department of Neurology, University of Michigan Health System, Ann

Arbor, MI 48109-0316, USA.

Sensory polyneuropathy can be a serious problem, but for the majority

of clinically important neuropathies there are no available

therapies. Neurotrophic and neuroprotective peptide factors have been

identified that prevent or reverse neuropathy in rodent models of

disease, but delivery of these highly pleiotropic peptides has posed

an obstacle for translation into effective human therapies. Gene

transfer into muscle using viral or non-viral vectors, or into

neurons of the dorsal root ganglion using herpes simplex virus-based

vectors, provides an alternative means to achieve this end. Studies

in animal models have been promising, and the first human trial,

using a plasmid to transfer the gene coding for vascular endothelial

growth factor into muscle for the treatment of diabetic neuropathy,

is now underway. Evidence supporting the trial and the challenges

facing this therapy are reviewed.