Gene therapy for Parkinson's disease

Show simple item record Lawlor, P.A. en During, M.J. en 2009-09-07T01:21:07Z en 2009-09-07T01:21:07Z en 2004 en
dc.identifier.citation Expert Reviews in Molecular Medicine 6 (5), 18. 2004 en
dc.identifier.issn 1462-3994 en
dc.identifier.other eid=2-s2.0-23844448882 en
dc.identifier.uri en
dc.description.abstract Parkinson's disease (PD) is a debilitating neurodegenerative disorder arising from loss of dopaminergic neurons in the substantia nigra pars compacta and subsequent depletion of striatal dopamine levels, which results in distressing motor symptoms. The current standard pharmacological treatment for PD is direct replacement of dopamine by treatment with its precursor, levodopa (L-dopa). However, this does not significantly alter disease progression and might contribute to the ongoing pathology. Several features of PD make this disease one of the most promising targets for clinical gene therapy of any neurological disease. The confinement of the major pathology to a compact, localised neuronal population and the anatomy of the basal ganglia circuitry mean that global gene transfer is not required and there are well-defined sites for gene transfer. The multifactorial aetiology of idiopathic PD means that it is unlikely any single gene will cure the disease, and as a result at least three separate gene-transfer strategies are currently being pursued: transfer of genes for enzymes involved in dopamine production; transfer of genes for growth factors involved in dopaminergic cell survival and regeneration; and transfer of genes to reset neuronal circuitry by switching cellular phenotype. The merits of these strategies are discussed here, along with remaining hurdles that might impede transfer of gene therapy technology to the clinic as a treatment for PD. en
dc.publisher Cambridge University Press en
dc.relation.ispartofseries Expert Reviews in Molecular Medicine en
dc.rights Items in ResearchSpace are protected by copyright, with all rights reserved, unless otherwise indicated. Previously published items are made available in accordance with the copyright policy of the publisher. Details obtained from en
dc.rights.uri en
dc.source.uri en
dc.title Gene therapy for Parkinson's disease en
dc.type Journal Article en
dc.identifier.doi 10.1017/S146239940400746X en
pubs.issue 5 en
pubs.begin-page 18 en
pubs.volume 6 en
dc.rights.holder Copyright: Cambridge University Press. en
dc.identifier.pmid 15000692 en
dc.rights.accessrights en

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