TARA: Scientific rationale for the development of gene therapy strategies for Parkinson’s disease

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Please use this identifier to cite or link to this item: http://hdl.handle.net/2262/42746

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Title:	Scientific rationale for the development of gene therapy strategies for Parkinson’s disease
Keywords:	6-OHDA6-hydroxydopamine AADCaromatic acid decarboxylase COMTcatechol-O-methyltransferase DBSdeep brain stimulation DAdopamine DATdopamine transporter EIAVequine infectious anemia virus FDGfluorodeoxyglucose FMTfluoro-L-m-tyrosine GABAgamma-aminobutyric acid GDNFglial derived neurotrophic factor GFRglycosyl-phos-phatidylinositol (GPI)-linked subunit GPglobus pallidus GPiinternal segment of globus pallidus Gluglutamate GADglutamic acid decarboxylase GFPGreen Fluorescent Protein GCH1GTP cyclohydrolase 1 ICVintracerebroventricular IgSPimmunoglobulin heavy-chain signal peptide L-DOPA3,4-dihydroxyphenylalanine MPTP1-methyl 4-phenyl 1,2,3,6-tetrahydropyridine NGFnerve growth factor NTNneurturin PDParkinson’s disease PDCPParkinson’s disease-related cognitive pattern PDRPParkinson’s disease-related covariance pattern PPRSPrimate Parkinsonian Rating Scale PETPositron Emission Tomography rAAVrecombinant adenoassociated virus rAdrecombinant adenovirus rLVrecombinant lentivirus BH4tetrahydrobiopterin 5HTserotonin STNsubthalamic nucleus SNrsubstantia nigra pars reticulate THtyrosine hydroxylase UPDRSUnified Parkinson’s Disease Rating Scale VGvector genomes viral vectors in vivo gene transfer neurotrophic factors neurorestoration neuroprotection enzyme replacement dopamine tyrosine hydroxylas
Issue Date:	15-Jun-2009
Publisher:	Elsevier
Abstract:	Abstract The ever-evolving understanding of the neuronal systems involved in Parkinson's disease together with the recent advances in recombinant viral vector technology has lead to the development of several gene therapy applications that are now entering into clinical testing phase. To date, four fundamentally different approaches have been pursued utilizing recombinant adeno-associated virus and lentiviruses as vectors for delivery. These strategies aim either to restore the lost brain functions by substitution of enzymes critical for synthesis of neurotransmitters or neurotrophic factors as a means to boost the function of remaining neurons in the diseased brain. In this review we discuss the differences in mechanism of action and describe the scientific rationale behind the currently tested gene therapy approaches for Parkinson’s disease in some detail and pinpoint their individual unique strengths and weaknesses
URI:	http://hdl.handle.net/2262/42746
DOI:	10.1016/j.bbadis.2009.02.009
Rights:	2009
Affiliation:	Brain Repair And Imaging in Neural Systems--> , Department of Experimental Medical Science--> , Lund University--> , Lund--> - SWEDEN (Björklund, Tomas) Brain Repair And Imaging in Neural Systems--> , Department of Experimental Medical Science--> , Lund University--> , Lund--> - SWEDEN (Kirik, Deniz) Lund University Bioimaging Center--> , Lund--> - SWEDEN (Kirik, Deniz) Brain Repair And Imaging in Neural Systems--> , Department of Experimental Medical Science--> , Lund University--> , Lund--> - SWEDEN (Kirik, Deniz)
Appears in Collections:	PEER Publications

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