Safe, Efficient, and Reproducible Gene Therapy of the Brain in the Dog Models of Sanfilippo and Hurler Syndromes
Identifieur interne : 000C58 ( Ncbi/Curation ); précédent : 000C57; suivant : 000C59Safe, Efficient, and Reproducible Gene Therapy of the Brain in the Dog Models of Sanfilippo and Hurler Syndromes
Auteurs : N Matthew Ellinwood [États-Unis] ; Jérôme Ausseil [France] ; Nathalie Desmaris [France] ; Stéphanie Bigou [France] ; Song Liu [France] ; Jackie K. Jens [États-Unis] ; Elizabeth M. Snella [États-Unis] ; Eman Ea Mohammed [États-Unis] ; Christopher B. Thomson [États-Unis] ; Sylvie Raoul [France] ; Béatrice Joussemet [France] ; Françoise Roux [France] ; Yan Chérel [France] ; Yaouen Lajat [France] ; Monique Piraud [France] ; Rachid Benchaouir [Pays-Bas] ; Stephan Hermening [Pays-Bas] ; Harald Petry [Pays-Bas] ; Roseline Froissart [France] ; Marc Tardieu [France] ; Carine Ciron [France] ; Philippe Moullier [France] ; Jennifer Parkes [États-Unis] ; Karen L. Kline [États-Unis] ; Irène Maire [France] ; Marie-Thérèse Vanier [France] ; Jean-Michel Heard [France] ; Marie-Anne Colle [France]Source :
- Molecular Therapy [ 1525-0016 ] ; 2010.
Abstract
Recent trials in patients with neurodegenerative diseases documented the safety of gene therapy based on adeno-associated virus (AAV) vectors deposited into the brain. Inborn errors of the metabolism are the most frequent causes of neurodegeneration in pre-adulthood. In Sanfilippo syndrome, a lysosomal storage disease in which heparan sulfate oligosaccharides accumulate, the onset of clinical manifestation is before 5 years. Studies in the mouse model showed that gene therapy providing the missing enzyme α-
Url:
DOI: 10.1038/mt.2010.265
PubMed: 21139569
PubMed Central: 3034858
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PMC:3034858Le document en format XML
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</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author><name sortKey="Snella, Elizabeth M" sort="Snella, Elizabeth M" uniqKey="Snella E" first="Elizabeth M" last="Snella">Elizabeth M. Snella</name>
<affiliation wicri:level="1"><nlm:aff id="aff1"><institution>Department of Animal Science and the Center for Integrated Animal Genomics, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author><name sortKey="Mohammed, Eman Ea" sort="Mohammed, Eman Ea" uniqKey="Mohammed E" first="Eman Ea" last="Mohammed">Eman Ea Mohammed</name>
<affiliation wicri:level="1"><nlm:aff id="aff1"><institution>Department of Animal Science and the Center for Integrated Animal Genomics, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author><name sortKey="Thomson, Christopher B" sort="Thomson, Christopher B" uniqKey="Thomson C" first="Christopher B" last="Thomson">Christopher B. Thomson</name>
<affiliation wicri:level="1"><nlm:aff id="aff1"><institution>Department of Animal Science and the Center for Integrated Animal Genomics, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author><name sortKey="Raoul, Sylvie" sort="Raoul, Sylvie" uniqKey="Raoul S" first="Sylvie" last="Raoul">Sylvie Raoul</name>
<affiliation wicri:level="1"><nlm:aff id="aff5"><institution>Service de Neurochirurgie, CHU Nord</institution>
, Nantes,<country>France</country>
</nlm:aff>
<country xml:lang="fr">France</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author><name sortKey="Joussemet, Beatrice" sort="Joussemet, Beatrice" uniqKey="Joussemet B" first="Béatrice" last="Joussemet">Béatrice Joussemet</name>
<affiliation wicri:level="1"><nlm:aff id="aff6"><institution>Centre de Boisbonne, Ecole Nationale Vétérinaire, Agroalimentaire et de l'Alimentation Nantes-Atlantiques (Oniris)</institution>
, Nantes,<country>France</country>
</nlm:aff>
<country xml:lang="fr">France</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author><name sortKey="Roux, Francoise" sort="Roux, Francoise" uniqKey="Roux F" first="Françoise" last="Roux">Françoise Roux</name>
<affiliation wicri:level="1"><nlm:aff id="aff6"><institution>Centre de Boisbonne, Ecole Nationale Vétérinaire, Agroalimentaire et de l'Alimentation Nantes-Atlantiques (Oniris)</institution>
, Nantes,<country>France</country>
</nlm:aff>
<country xml:lang="fr">France</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author><name sortKey="Cherel, Yan" sort="Cherel, Yan" uniqKey="Cherel Y" first="Yan" last="Chérel">Yan Chérel</name>
<affiliation wicri:level="1"><nlm:aff id="aff7"><institution>UMR INRA 703, Ecole Nationale Vétérinaire, Agroalimentaire et de l'Alimentation Nantes-Atlantiques (Oniris)</institution>
, Nantes,<country>France</country>
</nlm:aff>
<country xml:lang="fr">France</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author><name sortKey="Lajat, Yaouen" sort="Lajat, Yaouen" uniqKey="Lajat Y" first="Yaouen" last="Lajat">Yaouen Lajat</name>
<affiliation wicri:level="1"><nlm:aff id="aff5"><institution>Service de Neurochirurgie, CHU Nord</institution>
, Nantes,<country>France</country>
</nlm:aff>
<country xml:lang="fr">France</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author><name sortKey="Piraud, Monique" sort="Piraud, Monique" uniqKey="Piraud M" first="Monique" last="Piraud">Monique Piraud</name>
<affiliation wicri:level="1"><nlm:aff id="aff8"><institution>Maladies Héréditaire du Métabolisme et Dépistage Néonatal, Centre de Biologie et de Pathologie, Groupement Hospitalier Est, CBPE</institution>
, Bron,<country>France</country>
</nlm:aff>
<country xml:lang="fr">France</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author><name sortKey="Benchaouir, Rachid" sort="Benchaouir, Rachid" uniqKey="Benchaouir R" first="Rachid" last="Benchaouir">Rachid Benchaouir</name>
<affiliation wicri:level="1"><nlm:aff id="aff9"><institution>Amsterdam Molecular Therapeutics (AMT)</institution>
, Amsterdam,<country>The Netherlands</country>
</nlm:aff>
<country xml:lang="fr">Pays-Bas</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author><name sortKey="Hermening, Stephan" sort="Hermening, Stephan" uniqKey="Hermening S" first="Stephan" last="Hermening">Stephan Hermening</name>
<affiliation wicri:level="1"><nlm:aff id="aff9"><institution>Amsterdam Molecular Therapeutics (AMT)</institution>
, Amsterdam,<country>The Netherlands</country>
</nlm:aff>
<country xml:lang="fr">Pays-Bas</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author><name sortKey="Petry, Harald" sort="Petry, Harald" uniqKey="Petry H" first="Harald" last="Petry">Harald Petry</name>
<affiliation wicri:level="1"><nlm:aff id="aff9"><institution>Amsterdam Molecular Therapeutics (AMT)</institution>
, Amsterdam,<country>The Netherlands</country>
</nlm:aff>
<country xml:lang="fr">Pays-Bas</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author><name sortKey="Froissart, Roseline" sort="Froissart, Roseline" uniqKey="Froissart R" first="Roseline" last="Froissart">Roseline Froissart</name>
<affiliation wicri:level="1"><nlm:aff id="aff8"><institution>Maladies Héréditaire du Métabolisme et Dépistage Néonatal, Centre de Biologie et de Pathologie, Groupement Hospitalier Est, CBPE</institution>
, Bron,<country>France</country>
</nlm:aff>
<country xml:lang="fr">France</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author><name sortKey="Tardieu, Marc" sort="Tardieu, Marc" uniqKey="Tardieu M" first="Marc" last="Tardieu">Marc Tardieu</name>
<affiliation wicri:level="1"><nlm:aff id="aff10"><institution>Service de Neurologie Pédiatrique, Hôpital Bicêtre, Assistance Publique/Hôpitaux de Paris, INSERM U109</institution>
, le Kremlin-Bicêtre,<country>France</country>
</nlm:aff>
<country xml:lang="fr">France</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author><name sortKey="Ciron, Carine" sort="Ciron, Carine" uniqKey="Ciron C" first="Carine" last="Ciron">Carine Ciron</name>
<affiliation wicri:level="1"><nlm:aff id="aff11"><institution>INSERM U649, Université de Nantes</institution>
, Nantes,<country>France</country>
</nlm:aff>
<country xml:lang="fr">France</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author><name sortKey="Moullier, Philippe" sort="Moullier, Philippe" uniqKey="Moullier P" first="Philippe" last="Moullier">Philippe Moullier</name>
<affiliation wicri:level="1"><nlm:aff id="aff11"><institution>INSERM U649, Université de Nantes</institution>
, Nantes,<country>France</country>
</nlm:aff>
<country xml:lang="fr">France</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author><name sortKey="Parkes, Jennifer" sort="Parkes, Jennifer" uniqKey="Parkes J" first="Jennifer" last="Parkes">Jennifer Parkes</name>
<affiliation wicri:level="1"><nlm:aff id="aff2"><institution>Department of Veterinary Clinical Science, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author><name sortKey="Kline, Karen L" sort="Kline, Karen L" uniqKey="Kline K" first="Karen L" last="Kline">Karen L. Kline</name>
<affiliation wicri:level="1"><nlm:aff id="aff2"><institution>Department of Veterinary Clinical Science, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author><name sortKey="Maire, Irene" sort="Maire, Irene" uniqKey="Maire I" first="Irène" last="Maire">Irène Maire</name>
<affiliation wicri:level="1"><nlm:aff id="aff8"><institution>Maladies Héréditaire du Métabolisme et Dépistage Néonatal, Centre de Biologie et de Pathologie, Groupement Hospitalier Est, CBPE</institution>
, Bron,<country>France</country>
</nlm:aff>
<country xml:lang="fr">France</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author><name sortKey="Vanier, Marie Therese" sort="Vanier, Marie Therese" uniqKey="Vanier M" first="Marie-Thérèse" last="Vanier">Marie-Thérèse Vanier</name>
<affiliation wicri:level="1"><nlm:aff id="aff12"><institution>INSERM U820, Faculté de Médecine Laënnec</institution>
, Lyon,<country>France</country>
</nlm:aff>
<country xml:lang="fr">France</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author><name sortKey="Heard, Jean Michel" sort="Heard, Jean Michel" uniqKey="Heard J" first="Jean-Michel" last="Heard">Jean-Michel Heard</name>
<affiliation wicri:level="1"><nlm:aff id="aff3"><institution>Institut Pasteur, Unité Rétrovirus et Transfert Génétique, Département de Neuroscience</institution>
, Paris,<country>France</country>
</nlm:aff>
<country xml:lang="fr">France</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
<affiliation wicri:level="1"><nlm:aff id="aff4"><institution>INSERM U622</institution>
, Paris,<country>France</country>
</nlm:aff>
<country xml:lang="fr">France</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
<author><name sortKey="Colle, Marie Anne" sort="Colle, Marie Anne" uniqKey="Colle M" first="Marie-Anne" last="Colle">Marie-Anne Colle</name>
<affiliation wicri:level="1"><nlm:aff id="aff7"><institution>UMR INRA 703, Ecole Nationale Vétérinaire, Agroalimentaire et de l'Alimentation Nantes-Atlantiques (Oniris)</institution>
, Nantes,<country>France</country>
</nlm:aff>
<country xml:lang="fr">France</country>
<wicri:regionArea># see nlm:aff country strict</wicri:regionArea>
</affiliation>
</author>
</analytic>
<series><title level="j">Molecular Therapy</title>
<idno type="ISSN">1525-0016</idno>
<idno type="eISSN">1525-0024</idno>
<imprint><date when="2010">2010</date>
</imprint>
</series>
</biblStruct>
</sourceDesc>
</fileDesc>
<profileDesc><textClass></textClass>
</profileDesc>
</teiHeader>
<front><div type="abstract" xml:lang="en"><p>Recent trials in patients with neurodegenerative diseases documented the safety of gene therapy based on adeno-associated virus (AAV) vectors deposited into the brain. Inborn errors of the metabolism are the most frequent causes of neurodegeneration in pre-adulthood. In Sanfilippo syndrome, a lysosomal storage disease in which heparan sulfate oligosaccharides accumulate, the onset of clinical manifestation is before 5 years. Studies in the mouse model showed that gene therapy providing the missing enzyme α-<italic>N</italic>
-acetyl-glucosaminidase to brain cells prevents neurodegeneration and improves behavior. We now document safety and efficacy in affected dogs. Animals received eight deposits of a serotype 5 AAV vector, including vector prepared in insect Sf9 cells. As shown previously in dogs with the closely related Hurler syndrome, immunosuppression was necessary to prevent neuroinflammation and elimination of transduced cells. In immunosuppressed dogs, vector was efficiently delivered throughout the brain, induced α-<italic>N</italic>
-acetyl-glucosaminidase production, cleared stored compounds and storage lesions. The suitability of the procedure for clinical application was further assessed in Hurler dogs, providing information on reproducibility, tolerance, appropriate vector type and dosage, and optimal age for treatment in a total number of 25 treated dogs. Results strongly support projects of human trials aimed at assessing this treatment in Sanfilippo syndrome.</p>
</div>
</front>
</TEI>
</record>
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