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  Gene therapy for Parkinson's disease: Disease modification by GDNF family of ligands

Kirik, D., Cederfjall, E., Halliday, G., & Petersen, A. (2017). Gene therapy for Parkinson's disease: Disease modification by GDNF family of ligands. Neurobiology of Disease, 97, 179-188. doi:10.1016/j.nbd.2016.09.008.

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Kirik, Deniz, Autor
Cederfjall, Erik1, Autor           
Halliday, Glenda, Autor
Petersen, Asa, Autor
Affiliations:
1Department: Molecules-Signaling-Development / Klein, MPI of Neurobiology, Max Planck Society, ou_1113546              

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Schlagwörter: RECOMBINANT ADENOASSOCIATED VIRUS; NIGROSTRIATAL DOPAMINE SYSTEM; NEUROTROPHIC FACTOR DELIVERY; INCIDENTAL LEWY BODY; ALPHA-SYNUCLEIN; TYROSINE-HYDROXYLASE; RAT MODEL; HUNTINGTONS-DISEASE; LENTIVIRAL VECTOR; AXONAL-TRANSPORTNeurosciences & Neurology; Adeno-associated virus; AAV; Viral vectors; Non-human primates; GDNF; Neurturin; Neurotrophic factors; Dopamine;
 Zusammenfassung: Gene transfer is a promising drug delivery method of advanced therapeutic entities for Parkinson's disease. One advantage over conventional therapies, such as peripheral delivery of the dopamine pre-cursor L-DOPA, is site specific expression of proteins with regenerative, disease-modifying and potentially neuroprotective capacity. Several clinical trials have been performed to test the capacity of glial-cell line derived neurotrophic factor and neurturin to rescue degenerating dopaminergic neurons in the substantia nigra and their axon terminals in the striatum by delivery of these neurotrophic factors either as purified protein or by means of viral vector mediated gene delivery to the brain. Although gene therapy approaches tested so far have been shown to be safe, none met their primary endpoints in phase II clinical trials designed and powered to test the efficacy of the intervention. Within the scope of this review we aim to describe the state-of-the-art in the field, how different technical parameters were translated from pre-clinical studies in non-human primates to clinical trials, and what these trials taught us regarding important factors that may pave the way to the success of gene therapy for the treatment of Parkinson's disease. (C) 2016 Elsevier Inc. All rights reserved.

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Sprache(n): eng - English
 Datum: 2017-01-01
 Publikationsstatus: Erschienen
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 Identifikatoren: ISI: 000390618500012
DOI: 10.1016/j.nbd.2016.09.008
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Titel: Neurobiology of Disease
  Andere : Neurobiol. Dis.
Genre der Quelle: Zeitschrift
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Ort, Verlag, Ausgabe: Oxford : Academic Press
Seiten: - Band / Heft: 97 Artikelnummer: - Start- / Endseite: 179 - 188 Identifikator: ISSN: 0969-9961
CoNE: https://pure.mpg.de/cone/journals/resource/954922649144