title: Targeted drug delivery to the peripheral nervous system using gene therapy.
abstract: Gene transfer to target delivery of neurotrophic factors to the primary sensory afferent for treatment of polyneuropathy, or of inhibitory neurotransmitters for relief of chronic pain, offers the possibility of a highly selective targeted release of bioactive molecules within the nervous system. Preclinical studies with non-replicating herpes simplex virus (HSV)-based vectors injected into the skin to transduce neurons in the dorsal root ganglion have demonstrated efficacy in reducing-pain related behaviors in animal models of inflammatory pain, neuropathic pain, and pain caused by cancer, and in preventing progression of sensory neuropathy caused by toxins, chemotherapeutic drugs or resulting from diabetes. Successful completion of the first phase 1 clinical trial of HSV-mediated gene transfer in patients with intractable pain from cancer has set the stage for further clinical trials of this approach.
authors.affiliation: Diamyd Inc., Pittsburgh, PA, USA.
authors.forename: Darren
authors.initials: D
authors.lastname: Wolfe
authors.forename: Marina
authors.initials: M
authors.lastname: Mata
authors.forename: David J
authors.initials: DJ
authors.lastname: Fink
mesh_terms: D000818:Animals; D059350:Chronic Pain / Q000235:genetics / Q000378:metabolism / Q000628:therapy; D002986:Clinical Trials as Topic; D003929:Diabetic Neuropathies / Q000235:genetics / Q000378:metabolism / Q000628:therapy; D004745:Enkephalins / Q000235:genetics / Q000378:metabolism; D015316:Genetic Therapy; D005822:Genetic Vectors; D006564:Herpesviridae / Q000235:genetics; D006801:Humans; D009369:Neoplasms / Q000150:complications / Q000503:physiopathology; D009414:Nerve Growth Factors / Q000235:genetics / Q000378:metabolism; D018377:Neurotransmitter Agents / Q000235:genetics / Q000378:metabolism; D010148:Intractable Pain / Q000209:etiology / Q000235:genetics / Q000378:metabolism / Q000628:therapy; D017933:Peripheral Nervous System / Q000378:metabolism*; D011115:Polyneuropathies / Q000235:genetics / Q000378:metabolism / Q000628:therapy; D011498:Protein Precursors / Q000235:genetics / Q000378:metabolism
chemical_list: D004745:Enkephalins; D009414:Nerve Growth Factors; D018377:Neurotransmitter Agents; D011498:Protein Precursors; C029992:proenkephalin