Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial.
Identifieur interne : 000931 ( Main/Exploration ); précédent : 000930; suivant : 000932Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial.
Auteurs : Stéphane Palfi [France] ; Jean Marc Gurruchaga [France] ; G Scott Ralph [Royaume-Uni] ; Helene Lepetit [France] ; Sonia Lavisse [France] ; Philip C. Buttery [Royaume-Uni] ; Colin Watts [Royaume-Uni] ; James Miskin [Royaume-Uni] ; Michelle Kelleher [Royaume-Uni] ; Sarah Deeley [Royaume-Uni] ; Hirokazu Iwamuro [France] ; Jean Pascal Lefaucheur [France] ; Claire Thiriez [France] ; Gilles Fenelon [France] ; Cherry Lucas [Royaume-Uni] ; Pierre Brugières [France] ; Inanna Gabriel [France] ; Kou Abhay [France] ; Xavier Drouot [France] ; Naoki Tani [France] ; Aurelie Kas [France] ; Bijan Ghaleh [France] ; Philippe Le Corvoisier [France] ; Patrice Dolphin [France] ; David P. Breen [Royaume-Uni] ; Sarah Mason [Royaume-Uni] ; Natalie Valle Guzman [Royaume-Uni] ; Nicholas D. Mazarakis [Royaume-Uni] ; Pippa A. Radcliffe [Royaume-Uni] ; Richard Harrop [Royaume-Uni] ; Susan M. Kingsman [Royaume-Uni] ; Olivier Rascol [France] ; Stuart Naylor [Royaume-Uni] ; Roger A. Barker [Royaume-Uni] ; Philippe Hantraye [France] ; Philippe Remy [France] ; Pierre Cesaro [France] ; Kyriacos A. Mitrophanous [Royaume-Uni]Source :
- Lancet (London, England) [ 1474-547X ] ; 2014.
English descriptors
- KwdEn :
- Aged, Antiparkinson Agents (administration & dosage), Antiparkinson Agents (adverse effects), Dopa Decarboxylase (genetics), Dopamine (biosynthesis), Dopaminergic Neurons (metabolism), Dopaminergic Neurons (virology), Follow-Up Studies, GTP Cyclohydrolase (administration & dosage), GTP Cyclohydrolase (adverse effects), GTP Cyclohydrolase (genetics), Genetic Therapy (adverse effects), Genetic Therapy (methods), Genetic Vectors (administration & dosage), Genetic Vectors (adverse effects), Humans, Infectious Anemia Virus, Equine (genetics), Injections, Intralesional, Male, Middle Aged, Parkinson Disease (therapy), Putamen, Transfection (methods), Transgenes (genetics), Tyrosine 3-Monooxygenase (administration & dosage), Tyrosine 3-Monooxygenase (adverse effects), Tyrosine 3-Monooxygenase (genetics).
- MESH :
- chemical , administration & dosage : Antiparkinson Agents, GTP Cyclohydrolase, Tyrosine 3-Monooxygenase.
- chemical , adverse effects : Antiparkinson Agents, GTP Cyclohydrolase, Tyrosine 3-Monooxygenase.
- chemical , biosynthesis : Dopamine.
- chemical , genetics : Dopa Decarboxylase, GTP Cyclohydrolase, Tyrosine 3-Monooxygenase.
- administration & dosage : Genetic Vectors.
- adverse effects : Genetic Therapy, Genetic Vectors.
- genetics : Infectious Anemia Virus, Equine, Transgenes.
- metabolism : Dopaminergic Neurons.
- methods : Genetic Therapy, Transfection.
- therapy : Parkinson Disease.
- virology : Dopaminergic Neurons.
- Aged, Follow-Up Studies, Humans, Injections, Intralesional, Male, Middle Aged, Putamen.
Abstract
Parkinson's disease is typically treated with oral dopamine replacement therapies; however, long-term treatment leads to motor complications and, occasionally, impulse control disorders caused by intermittent stimulation of dopamine receptors and off-target effects, respectively. We aimed to assess the safety, tolerability, and efficacy of bilateral, intrastriatal delivery of ProSavin, a lentiviral vector-based gene therapy aimed at restoring local and continuous dopamine production in patients with advanced Parkinson's disease.
DOI: 10.1016/S0140-6736(13)61939-X
PubMed: 24412048
Affiliations:
- France, Royaume-Uni
- Angleterre, Grand Londres, Midi-Pyrénées, Occitanie (région administrative), Oxfordshire, Île-de-France
- Créteil, Londres, Oxford, Toulouse
Links toward previous steps (curation, corpus...)
- to stream PubMed, to step Corpus: 000591
- to stream PubMed, to step Curation: 000571
- to stream PubMed, to step Checkpoint: 000571
- to stream Ncbi, to step Merge: 001305
- to stream Ncbi, to step Curation: 001305
- to stream Ncbi, to step Checkpoint: 001305
- to stream Main, to step Merge: 000941
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Le document en format XML
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<author><name sortKey="Iwamuro, Hirokazu" sort="Iwamuro, Hirokazu" uniqKey="Iwamuro H" first="Hirokazu" last="Iwamuro">Hirokazu Iwamuro</name>
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<author><name sortKey="Thiriez, Claire" sort="Thiriez, Claire" uniqKey="Thiriez C" first="Claire" last="Thiriez">Claire Thiriez</name>
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<author><name sortKey="Fenelon, Gilles" sort="Fenelon, Gilles" uniqKey="Fenelon G" first="Gilles" last="Fenelon">Gilles Fenelon</name>
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<author><name sortKey="Lucas, Cherry" sort="Lucas, Cherry" uniqKey="Lucas C" first="Cherry" last="Lucas">Cherry Lucas</name>
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<author><name sortKey="Brugieres, Pierre" sort="Brugieres, Pierre" uniqKey="Brugieres P" first="Pierre" last="Brugières">Pierre Brugières</name>
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<country xml:lang="fr">France</country>
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<author><name sortKey="Gabriel, Inanna" sort="Gabriel, Inanna" uniqKey="Gabriel I" first="Inanna" last="Gabriel">Inanna Gabriel</name>
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<author><name sortKey="Abhay, Kou" sort="Abhay, Kou" uniqKey="Abhay K" first="Kou" last="Abhay">Kou Abhay</name>
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<author><name sortKey="Drouot, Xavier" sort="Drouot, Xavier" uniqKey="Drouot X" first="Xavier" last="Drouot">Xavier Drouot</name>
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<author><name sortKey="Tani, Naoki" sort="Tani, Naoki" uniqKey="Tani N" first="Naoki" last="Tani">Naoki Tani</name>
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<author><name sortKey="Kas, Aurelie" sort="Kas, Aurelie" uniqKey="Kas A" first="Aurelie" last="Kas">Aurelie Kas</name>
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<author><name sortKey="Ghaleh, Bijan" sort="Ghaleh, Bijan" uniqKey="Ghaleh B" first="Bijan" last="Ghaleh">Bijan Ghaleh</name>
<affiliation wicri:level="3"><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation>
<country xml:lang="fr">France</country>
<wicri:regionArea>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil</wicri:regionArea>
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<author><name sortKey="Le Corvoisier, Philippe" sort="Le Corvoisier, Philippe" uniqKey="Le Corvoisier P" first="Philippe" last="Le Corvoisier">Philippe Le Corvoisier</name>
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<wicri:regionArea>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil</wicri:regionArea>
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</affiliation>
</author>
<author><name sortKey="Dolphin, Patrice" sort="Dolphin, Patrice" uniqKey="Dolphin P" first="Patrice" last="Dolphin">Patrice Dolphin</name>
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<country xml:lang="fr">France</country>
<wicri:regionArea>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil</wicri:regionArea>
<placeName><region type="region">Île-de-France</region>
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</affiliation>
</author>
<author><name sortKey="Breen, David P" sort="Breen, David P" uniqKey="Breen D" first="David P" last="Breen">David P. Breen</name>
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</affiliation>
</author>
<author><name sortKey="Mason, Sarah" sort="Mason, Sarah" uniqKey="Mason S" first="Sarah" last="Mason">Sarah Mason</name>
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<country xml:lang="fr">Royaume-Uni</country>
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</affiliation>
</author>
<author><name sortKey="Guzman, Natalie Valle" sort="Guzman, Natalie Valle" uniqKey="Guzman N" first="Natalie Valle" last="Guzman">Natalie Valle Guzman</name>
<affiliation wicri:level="1"><nlm:affiliation>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge, UK.</nlm:affiliation>
<country xml:lang="fr">Royaume-Uni</country>
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</author>
<author><name sortKey="Mazarakis, Nicholas D" sort="Mazarakis, Nicholas D" uniqKey="Mazarakis N" first="Nicholas D" last="Mazarakis">Nicholas D. Mazarakis</name>
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<country xml:lang="fr">Royaume-Uni</country>
<wicri:regionArea>Gene Therapy, Centre of Neuroinflammation and Neurodegeneration, Division of Brain Sciences, Faculty of Medicine, Hammersmith Hospital Campus, London</wicri:regionArea>
<placeName><settlement type="city">Londres</settlement>
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</affiliation>
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<author><name sortKey="Radcliffe, Pippa A" sort="Radcliffe, Pippa A" uniqKey="Radcliffe P" first="Pippa A" last="Radcliffe">Pippa A. Radcliffe</name>
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</placeName>
</affiliation>
</author>
<author><name sortKey="Harrop, Richard" sort="Harrop, Richard" uniqKey="Harrop R" first="Richard" last="Harrop">Richard Harrop</name>
<affiliation wicri:level="3"><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation>
<country xml:lang="fr">Royaume-Uni</country>
<wicri:regionArea>Oxford BioMedica, Oxford</wicri:regionArea>
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</placeName>
</affiliation>
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<author><name sortKey="Kingsman, Susan M" sort="Kingsman, Susan M" uniqKey="Kingsman S" first="Susan M" last="Kingsman">Susan M. Kingsman</name>
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<country xml:lang="fr">Royaume-Uni</country>
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<author><name sortKey="Rascol, Olivier" sort="Rascol, Olivier" uniqKey="Rascol O" first="Olivier" last="Rascol">Olivier Rascol</name>
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<country xml:lang="fr">France</country>
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<author><name sortKey="Naylor, Stuart" sort="Naylor, Stuart" uniqKey="Naylor S" first="Stuart" last="Naylor">Stuart Naylor</name>
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</placeName>
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<author><name sortKey="Barker, Roger A" sort="Barker, Roger A" uniqKey="Barker R" first="Roger A" last="Barker">Roger A. Barker</name>
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<country xml:lang="fr">Royaume-Uni</country>
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</author>
<author><name sortKey="Hantraye, Philippe" sort="Hantraye, Philippe" uniqKey="Hantraye P" first="Philippe" last="Hantraye">Philippe Hantraye</name>
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<country xml:lang="fr">France</country>
<wicri:regionArea>CEA, DSV I(2)BM, MIRCen and CNRS URA2210, Fontenay-aux-Roses</wicri:regionArea>
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<wicri:noRegion>Fontenay-aux-Roses</wicri:noRegion>
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</author>
<author><name sortKey="Remy, Philippe" sort="Remy, Philippe" uniqKey="Remy P" first="Philippe" last="Remy">Philippe Remy</name>
<affiliation wicri:level="1"><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France; CEA, DSV I(2)BM, MIRCen and CNRS URA2210, Fontenay-aux-Roses, France.</nlm:affiliation>
<country xml:lang="fr">France</country>
<wicri:regionArea>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France; CEA, DSV I(2)BM, MIRCen and CNRS URA2210, Fontenay-aux-Roses</wicri:regionArea>
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<author><name sortKey="Cesaro, Pierre" sort="Cesaro, Pierre" uniqKey="Cesaro P" first="Pierre" last="Cesaro">Pierre Cesaro</name>
<affiliation wicri:level="3"><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France; INSERM U955, E01, Institut de Recherche Biomédicale, Créteil, France.</nlm:affiliation>
<country xml:lang="fr">France</country>
<wicri:regionArea>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France; INSERM U955, E01, Institut de Recherche Biomédicale, Créteil</wicri:regionArea>
<placeName><region type="region">Île-de-France</region>
<region type="old region">Île-de-France</region>
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</placeName>
</affiliation>
</author>
<author><name sortKey="Mitrophanous, Kyriacos A" sort="Mitrophanous, Kyriacos A" uniqKey="Mitrophanous K" first="Kyriacos A" last="Mitrophanous">Kyriacos A. Mitrophanous</name>
<affiliation wicri:level="3"><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation>
<country xml:lang="fr">Royaume-Uni</country>
<wicri:regionArea>Oxford BioMedica, Oxford</wicri:regionArea>
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<sourceDesc><biblStruct><analytic><title xml:lang="en">Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial.</title>
<author><name sortKey="Palfi, Stephane" sort="Palfi, Stephane" uniqKey="Palfi S" first="Stéphane" last="Palfi">Stéphane Palfi</name>
<affiliation wicri:level="3"><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France. Electronic address: stephane.palfi@hmn.aphp.fr.</nlm:affiliation>
<country xml:lang="fr">France</country>
<wicri:regionArea>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil</wicri:regionArea>
<placeName><region type="region">Île-de-France</region>
<region type="old region">Île-de-France</region>
<settlement type="city">Créteil</settlement>
</placeName>
</affiliation>
</author>
<author><name sortKey="Gurruchaga, Jean Marc" sort="Gurruchaga, Jean Marc" uniqKey="Gurruchaga J" first="Jean Marc" last="Gurruchaga">Jean Marc Gurruchaga</name>
<affiliation wicri:level="3"><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation>
<country xml:lang="fr">France</country>
<wicri:regionArea>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil</wicri:regionArea>
<placeName><region type="region">Île-de-France</region>
<region type="old region">Île-de-France</region>
<settlement type="city">Créteil</settlement>
</placeName>
</affiliation>
</author>
<author><name sortKey="Ralph, G Scott" sort="Ralph, G Scott" uniqKey="Ralph G" first="G Scott" last="Ralph">G Scott Ralph</name>
<affiliation wicri:level="3"><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation>
<country xml:lang="fr">Royaume-Uni</country>
<wicri:regionArea>Oxford BioMedica, Oxford</wicri:regionArea>
<placeName><settlement type="city">Oxford</settlement>
<region type="country">Angleterre</region>
<region type="comté" nuts="2">Oxfordshire</region>
</placeName>
</affiliation>
</author>
<author><name sortKey="Lepetit, Helene" sort="Lepetit, Helene" uniqKey="Lepetit H" first="Helene" last="Lepetit">Helene Lepetit</name>
<affiliation wicri:level="3"><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation>
<country xml:lang="fr">France</country>
<wicri:regionArea>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil</wicri:regionArea>
<placeName><region type="region">Île-de-France</region>
<region type="old region">Île-de-France</region>
<settlement type="city">Créteil</settlement>
</placeName>
</affiliation>
</author>
<author><name sortKey="Lavisse, Sonia" sort="Lavisse, Sonia" uniqKey="Lavisse S" first="Sonia" last="Lavisse">Sonia Lavisse</name>
<affiliation wicri:level="1"><nlm:affiliation>CEA, DSV I(2)BM, MIRCen and CNRS URA2210, Fontenay-aux-Roses, France.</nlm:affiliation>
<country xml:lang="fr">France</country>
<wicri:regionArea>CEA, DSV I(2)BM, MIRCen and CNRS URA2210, Fontenay-aux-Roses</wicri:regionArea>
<wicri:noRegion>Fontenay-aux-Roses</wicri:noRegion>
<wicri:noRegion>Fontenay-aux-Roses</wicri:noRegion>
</affiliation>
</author>
<author><name sortKey="Buttery, Philip C" sort="Buttery, Philip C" uniqKey="Buttery P" first="Philip C" last="Buttery">Philip C. Buttery</name>
<affiliation wicri:level="1"><nlm:affiliation>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge, UK.</nlm:affiliation>
<country xml:lang="fr">Royaume-Uni</country>
<wicri:regionArea>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge</wicri:regionArea>
<wicri:noRegion>Cambridge</wicri:noRegion>
</affiliation>
</author>
<author><name sortKey="Watts, Colin" sort="Watts, Colin" uniqKey="Watts C" first="Colin" last="Watts">Colin Watts</name>
<affiliation wicri:level="1"><nlm:affiliation>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge, UK.</nlm:affiliation>
<country xml:lang="fr">Royaume-Uni</country>
<wicri:regionArea>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge</wicri:regionArea>
<wicri:noRegion>Cambridge</wicri:noRegion>
</affiliation>
</author>
<author><name sortKey="Miskin, James" sort="Miskin, James" uniqKey="Miskin J" first="James" last="Miskin">James Miskin</name>
<affiliation wicri:level="3"><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation>
<country xml:lang="fr">Royaume-Uni</country>
<wicri:regionArea>Oxford BioMedica, Oxford</wicri:regionArea>
<placeName><settlement type="city">Oxford</settlement>
<region type="country">Angleterre</region>
<region type="comté" nuts="2">Oxfordshire</region>
</placeName>
</affiliation>
</author>
<author><name sortKey="Kelleher, Michelle" sort="Kelleher, Michelle" uniqKey="Kelleher M" first="Michelle" last="Kelleher">Michelle Kelleher</name>
<affiliation wicri:level="3"><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation>
<country xml:lang="fr">Royaume-Uni</country>
<wicri:regionArea>Oxford BioMedica, Oxford</wicri:regionArea>
<placeName><settlement type="city">Oxford</settlement>
<region type="country">Angleterre</region>
<region type="comté" nuts="2">Oxfordshire</region>
</placeName>
</affiliation>
</author>
<author><name sortKey="Deeley, Sarah" sort="Deeley, Sarah" uniqKey="Deeley S" first="Sarah" last="Deeley">Sarah Deeley</name>
<affiliation wicri:level="3"><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation>
<country xml:lang="fr">Royaume-Uni</country>
<wicri:regionArea>Oxford BioMedica, Oxford</wicri:regionArea>
<placeName><settlement type="city">Oxford</settlement>
<region type="country">Angleterre</region>
<region type="comté" nuts="2">Oxfordshire</region>
</placeName>
</affiliation>
</author>
<author><name sortKey="Iwamuro, Hirokazu" sort="Iwamuro, Hirokazu" uniqKey="Iwamuro H" first="Hirokazu" last="Iwamuro">Hirokazu Iwamuro</name>
<affiliation wicri:level="3"><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation>
<country xml:lang="fr">France</country>
<wicri:regionArea>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil</wicri:regionArea>
<placeName><region type="region">Île-de-France</region>
<region type="old region">Île-de-France</region>
<settlement type="city">Créteil</settlement>
</placeName>
</affiliation>
</author>
<author><name sortKey="Lefaucheur, Jean Pascal" sort="Lefaucheur, Jean Pascal" uniqKey="Lefaucheur J" first="Jean Pascal" last="Lefaucheur">Jean Pascal Lefaucheur</name>
<affiliation wicri:level="3"><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation>
<country xml:lang="fr">France</country>
<wicri:regionArea>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil</wicri:regionArea>
<placeName><region type="region">Île-de-France</region>
<region type="old region">Île-de-France</region>
<settlement type="city">Créteil</settlement>
</placeName>
</affiliation>
</author>
<author><name sortKey="Thiriez, Claire" sort="Thiriez, Claire" uniqKey="Thiriez C" first="Claire" last="Thiriez">Claire Thiriez</name>
<affiliation wicri:level="3"><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation>
<country xml:lang="fr">France</country>
<wicri:regionArea>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil</wicri:regionArea>
<placeName><region type="region">Île-de-France</region>
<region type="old region">Île-de-France</region>
<settlement type="city">Créteil</settlement>
</placeName>
</affiliation>
</author>
<author><name sortKey="Fenelon, Gilles" sort="Fenelon, Gilles" uniqKey="Fenelon G" first="Gilles" last="Fenelon">Gilles Fenelon</name>
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<country xml:lang="fr">France</country>
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<country xml:lang="fr">France</country>
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<country xml:lang="fr">France</country>
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</affiliation>
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<author><name sortKey="Abhay, Kou" sort="Abhay, Kou" uniqKey="Abhay K" first="Kou" last="Abhay">Kou Abhay</name>
<affiliation wicri:level="3"><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation>
<country xml:lang="fr">France</country>
<wicri:regionArea>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil</wicri:regionArea>
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<author><name sortKey="Drouot, Xavier" sort="Drouot, Xavier" uniqKey="Drouot X" first="Xavier" last="Drouot">Xavier Drouot</name>
<affiliation wicri:level="3"><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation>
<country xml:lang="fr">France</country>
<wicri:regionArea>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil</wicri:regionArea>
<placeName><region type="region">Île-de-France</region>
<region type="old region">Île-de-France</region>
<settlement type="city">Créteil</settlement>
</placeName>
</affiliation>
</author>
<author><name sortKey="Tani, Naoki" sort="Tani, Naoki" uniqKey="Tani N" first="Naoki" last="Tani">Naoki Tani</name>
<affiliation wicri:level="3"><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation>
<country xml:lang="fr">France</country>
<wicri:regionArea>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil</wicri:regionArea>
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<settlement type="city">Créteil</settlement>
</placeName>
</affiliation>
</author>
<author><name sortKey="Kas, Aurelie" sort="Kas, Aurelie" uniqKey="Kas A" first="Aurelie" last="Kas">Aurelie Kas</name>
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<author><name sortKey="Ghaleh, Bijan" sort="Ghaleh, Bijan" uniqKey="Ghaleh B" first="Bijan" last="Ghaleh">Bijan Ghaleh</name>
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<country xml:lang="fr">France</country>
<wicri:regionArea>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil</wicri:regionArea>
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<author><name sortKey="Le Corvoisier, Philippe" sort="Le Corvoisier, Philippe" uniqKey="Le Corvoisier P" first="Philippe" last="Le Corvoisier">Philippe Le Corvoisier</name>
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<country xml:lang="fr">France</country>
<wicri:regionArea>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil</wicri:regionArea>
<placeName><region type="region">Île-de-France</region>
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<author><name sortKey="Dolphin, Patrice" sort="Dolphin, Patrice" uniqKey="Dolphin P" first="Patrice" last="Dolphin">Patrice Dolphin</name>
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<country xml:lang="fr">France</country>
<wicri:regionArea>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil</wicri:regionArea>
<placeName><region type="region">Île-de-France</region>
<region type="old region">Île-de-France</region>
<settlement type="city">Créteil</settlement>
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<author><name sortKey="Breen, David P" sort="Breen, David P" uniqKey="Breen D" first="David P" last="Breen">David P. Breen</name>
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<author><name sortKey="Mason, Sarah" sort="Mason, Sarah" uniqKey="Mason S" first="Sarah" last="Mason">Sarah Mason</name>
<affiliation wicri:level="1"><nlm:affiliation>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge, UK.</nlm:affiliation>
<country xml:lang="fr">Royaume-Uni</country>
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<author><name sortKey="Guzman, Natalie Valle" sort="Guzman, Natalie Valle" uniqKey="Guzman N" first="Natalie Valle" last="Guzman">Natalie Valle Guzman</name>
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<author><name sortKey="Mazarakis, Nicholas D" sort="Mazarakis, Nicholas D" uniqKey="Mazarakis N" first="Nicholas D" last="Mazarakis">Nicholas D. Mazarakis</name>
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<country xml:lang="fr">Royaume-Uni</country>
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<country xml:lang="fr">Royaume-Uni</country>
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<country xml:lang="fr">France</country>
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<author><name sortKey="Barker, Roger A" sort="Barker, Roger A" uniqKey="Barker R" first="Roger A" last="Barker">Roger A. Barker</name>
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<country xml:lang="fr">Royaume-Uni</country>
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<country xml:lang="fr">France</country>
<wicri:regionArea>CEA, DSV I(2)BM, MIRCen and CNRS URA2210, Fontenay-aux-Roses</wicri:regionArea>
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<country xml:lang="fr">France</country>
<wicri:regionArea>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France; CEA, DSV I(2)BM, MIRCen and CNRS URA2210, Fontenay-aux-Roses</wicri:regionArea>
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<country xml:lang="fr">France</country>
<wicri:regionArea>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France; INSERM U955, E01, Institut de Recherche Biomédicale, Créteil</wicri:regionArea>
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<term>Genetic Vectors (adverse effects)</term>
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<front><div type="abstract" xml:lang="en">Parkinson's disease is typically treated with oral dopamine replacement therapies; however, long-term treatment leads to motor complications and, occasionally, impulse control disorders caused by intermittent stimulation of dopamine receptors and off-target effects, respectively. We aimed to assess the safety, tolerability, and efficacy of bilateral, intrastriatal delivery of ProSavin, a lentiviral vector-based gene therapy aimed at restoring local and continuous dopamine production in patients with advanced Parkinson's disease.</div>
</front>
</TEI>
<affiliations><list><country><li>France</li>
<li>Royaume-Uni</li>
</country>
<region><li>Angleterre</li>
<li>Grand Londres</li>
<li>Midi-Pyrénées</li>
<li>Occitanie (région administrative)</li>
<li>Oxfordshire</li>
<li>Île-de-France</li>
</region>
<settlement><li>Créteil</li>
<li>Londres</li>
<li>Oxford</li>
<li>Toulouse</li>
</settlement>
</list>
<tree><country name="France"><region name="Île-de-France"><name sortKey="Palfi, Stephane" sort="Palfi, Stephane" uniqKey="Palfi S" first="Stéphane" last="Palfi">Stéphane Palfi</name>
</region>
<name sortKey="Abhay, Kou" sort="Abhay, Kou" uniqKey="Abhay K" first="Kou" last="Abhay">Kou Abhay</name>
<name sortKey="Brugieres, Pierre" sort="Brugieres, Pierre" uniqKey="Brugieres P" first="Pierre" last="Brugières">Pierre Brugières</name>
<name sortKey="Cesaro, Pierre" sort="Cesaro, Pierre" uniqKey="Cesaro P" first="Pierre" last="Cesaro">Pierre Cesaro</name>
<name sortKey="Dolphin, Patrice" sort="Dolphin, Patrice" uniqKey="Dolphin P" first="Patrice" last="Dolphin">Patrice Dolphin</name>
<name sortKey="Drouot, Xavier" sort="Drouot, Xavier" uniqKey="Drouot X" first="Xavier" last="Drouot">Xavier Drouot</name>
<name sortKey="Fenelon, Gilles" sort="Fenelon, Gilles" uniqKey="Fenelon G" first="Gilles" last="Fenelon">Gilles Fenelon</name>
<name sortKey="Gabriel, Inanna" sort="Gabriel, Inanna" uniqKey="Gabriel I" first="Inanna" last="Gabriel">Inanna Gabriel</name>
<name sortKey="Ghaleh, Bijan" sort="Ghaleh, Bijan" uniqKey="Ghaleh B" first="Bijan" last="Ghaleh">Bijan Ghaleh</name>
<name sortKey="Gurruchaga, Jean Marc" sort="Gurruchaga, Jean Marc" uniqKey="Gurruchaga J" first="Jean Marc" last="Gurruchaga">Jean Marc Gurruchaga</name>
<name sortKey="Hantraye, Philippe" sort="Hantraye, Philippe" uniqKey="Hantraye P" first="Philippe" last="Hantraye">Philippe Hantraye</name>
<name sortKey="Iwamuro, Hirokazu" sort="Iwamuro, Hirokazu" uniqKey="Iwamuro H" first="Hirokazu" last="Iwamuro">Hirokazu Iwamuro</name>
<name sortKey="Kas, Aurelie" sort="Kas, Aurelie" uniqKey="Kas A" first="Aurelie" last="Kas">Aurelie Kas</name>
<name sortKey="Lavisse, Sonia" sort="Lavisse, Sonia" uniqKey="Lavisse S" first="Sonia" last="Lavisse">Sonia Lavisse</name>
<name sortKey="Le Corvoisier, Philippe" sort="Le Corvoisier, Philippe" uniqKey="Le Corvoisier P" first="Philippe" last="Le Corvoisier">Philippe Le Corvoisier</name>
<name sortKey="Lefaucheur, Jean Pascal" sort="Lefaucheur, Jean Pascal" uniqKey="Lefaucheur J" first="Jean Pascal" last="Lefaucheur">Jean Pascal Lefaucheur</name>
<name sortKey="Lepetit, Helene" sort="Lepetit, Helene" uniqKey="Lepetit H" first="Helene" last="Lepetit">Helene Lepetit</name>
<name sortKey="Rascol, Olivier" sort="Rascol, Olivier" uniqKey="Rascol O" first="Olivier" last="Rascol">Olivier Rascol</name>
<name sortKey="Remy, Philippe" sort="Remy, Philippe" uniqKey="Remy P" first="Philippe" last="Remy">Philippe Remy</name>
<name sortKey="Tani, Naoki" sort="Tani, Naoki" uniqKey="Tani N" first="Naoki" last="Tani">Naoki Tani</name>
<name sortKey="Thiriez, Claire" sort="Thiriez, Claire" uniqKey="Thiriez C" first="Claire" last="Thiriez">Claire Thiriez</name>
</country>
<country name="Royaume-Uni"><region name="Angleterre"><name sortKey="Ralph, G Scott" sort="Ralph, G Scott" uniqKey="Ralph G" first="G Scott" last="Ralph">G Scott Ralph</name>
</region>
<name sortKey="Barker, Roger A" sort="Barker, Roger A" uniqKey="Barker R" first="Roger A" last="Barker">Roger A. Barker</name>
<name sortKey="Breen, David P" sort="Breen, David P" uniqKey="Breen D" first="David P" last="Breen">David P. Breen</name>
<name sortKey="Buttery, Philip C" sort="Buttery, Philip C" uniqKey="Buttery P" first="Philip C" last="Buttery">Philip C. Buttery</name>
<name sortKey="Deeley, Sarah" sort="Deeley, Sarah" uniqKey="Deeley S" first="Sarah" last="Deeley">Sarah Deeley</name>
<name sortKey="Guzman, Natalie Valle" sort="Guzman, Natalie Valle" uniqKey="Guzman N" first="Natalie Valle" last="Guzman">Natalie Valle Guzman</name>
<name sortKey="Harrop, Richard" sort="Harrop, Richard" uniqKey="Harrop R" first="Richard" last="Harrop">Richard Harrop</name>
<name sortKey="Kelleher, Michelle" sort="Kelleher, Michelle" uniqKey="Kelleher M" first="Michelle" last="Kelleher">Michelle Kelleher</name>
<name sortKey="Kingsman, Susan M" sort="Kingsman, Susan M" uniqKey="Kingsman S" first="Susan M" last="Kingsman">Susan M. Kingsman</name>
<name sortKey="Lucas, Cherry" sort="Lucas, Cherry" uniqKey="Lucas C" first="Cherry" last="Lucas">Cherry Lucas</name>
<name sortKey="Mason, Sarah" sort="Mason, Sarah" uniqKey="Mason S" first="Sarah" last="Mason">Sarah Mason</name>
<name sortKey="Mazarakis, Nicholas D" sort="Mazarakis, Nicholas D" uniqKey="Mazarakis N" first="Nicholas D" last="Mazarakis">Nicholas D. Mazarakis</name>
<name sortKey="Miskin, James" sort="Miskin, James" uniqKey="Miskin J" first="James" last="Miskin">James Miskin</name>
<name sortKey="Mitrophanous, Kyriacos A" sort="Mitrophanous, Kyriacos A" uniqKey="Mitrophanous K" first="Kyriacos A" last="Mitrophanous">Kyriacos A. Mitrophanous</name>
<name sortKey="Naylor, Stuart" sort="Naylor, Stuart" uniqKey="Naylor S" first="Stuart" last="Naylor">Stuart Naylor</name>
<name sortKey="Radcliffe, Pippa A" sort="Radcliffe, Pippa A" uniqKey="Radcliffe P" first="Pippa A" last="Radcliffe">Pippa A. Radcliffe</name>
<name sortKey="Watts, Colin" sort="Watts, Colin" uniqKey="Watts C" first="Colin" last="Watts">Colin Watts</name>
</country>
</tree>
</affiliations>
</record>
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