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Disease Modificatio...
Disease Modification by Combinatorial Single Vector Gene Therapy : A Preclinical Translational Study in Epilepsy
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- Melin, Esbjörn (författare)
- Lund University,Lunds universitet,Experimentell Epilepsi,Forskargrupper vid Lunds universitet,Experimental Epilepsy Group,Lund University Research Groups,Skåne University Hospital
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- Nanobashvili, Avtandil (författare)
- Lund University,Lunds universitet,Experimentell Epilepsi,Forskargrupper vid Lunds universitet,Experimental Epilepsy Group,Lund University Research Groups,CombiGene AB,Skåne University Hospital
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- Avdic, Una (författare)
- Lund University,Lunds universitet,Experimentell Epilepsi,Forskargrupper vid Lunds universitet,Experimental Epilepsy Group,Lund University Research Groups,Skåne University Hospital
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- Gøtzsche, Casper R. (författare)
- CombiGene AB,University of Copenhagen
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- Andersson, My (författare)
- Lund University,Lunds universitet,Epilepsicentrum,Sektion IV,Institutionen för kliniska vetenskaper, Lund,Medicinska fakulteten,Cellulär Neurofysiologi och Epilepsi,Forskargrupper vid Lunds universitet,Epilepsy Center,Section IV,Department of Clinical Sciences, Lund,Faculty of Medicine,Cellular Neurophysiology and Epilepsy group,Lund University Research Groups
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- Woldbye, David P.D. (författare)
- University of Copenhagen
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- Kokaia, Merab (författare)
- Lund University,Lunds universitet,NanoLund: Centre for Nanoscience,Annan verksamhet, LTH,Lunds Tekniska Högskola,Experimentell Epilepsi,Forskargrupper vid Lunds universitet,Other operations, LTH,Faculty of Engineering, LTH,Experimental Epilepsy Group,Lund University Research Groups,Skåne University Hospital
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(creator_code:org_t)
- Elsevier BV, 2019
- 2019
- Engelska 15 s.
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Ingår i: Molecular Therapy - Methods and Clinical Development. - : Elsevier BV. - 2329-0501. ; 15, s. 179-193
- Relaterad länk:
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http://dx.doi.org/10... (free)
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http://www.cell.com/...
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https://lup.lub.lu.s...
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https://doi.org/10.1...
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Abstract
Ämnesord
Stäng
- Gene therapy has been suggested as a plausible novel approach to achieve seizure control in patients with focal epilepsy that do not adequately respond to pharmacological treatment. We investigated the seizure-suppressant potential of combinatorial neuropeptide Y and Y2 receptor single vector gene therapy based on adeno-associated virus serotype 1 (AAV1) in rats. First, a dose-response study in the systemic kainate-induced acute seizure model was performed, whereby the 1012 genomic particles (gp)/mL titer of the vector was selected as an optimal concentration. Second, an efficacy study was performed in the intrahippocampal kainate chronic model of spontaneous recurrent seizures (SRSs), designed to reflect a likely clinical scenario, with magnetic resonance image (MRI)-guided focal unilateral administration of the vector in the hippocampus during the chronic stage of the disease. The efficacy study demonstrated a favorable outcome of the gene therapy, with a 31% responder rate (more than 50% reduction in SRS frequency) and 13% seizure-freedom rate, whereas no such effects were observed in the control animals. The inter-SRS and SRS cluster intervals were also significantly prolonged in the treated group compared to controls. In addition, the SRS duration was significantly reduced in the treated group but not in the controls. This study establishes the SRS-suppressant ability of the single vector combinatorial neuropeptide Y/Y2 receptor gene therapy in a clinically relevant chronic model of epilepsy.
Ämnesord
- MEDICIN OCH HÄLSOVETENSKAP -- Medicinska och farmaceutiska grundvetenskaper -- Neurovetenskaper (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Basic Medicine -- Neurosciences (hsv//eng)
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- art (ämneskategori)
- ref (ämneskategori)
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