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Journal articles
Manufacturing and Characterization of a Recombinant Adeno-Associated Virus Type 8 Reference Standard Material
Eduard Ayuso
1
Veronique Blouin
2, 3
Martin Lock
4
Susan Mcgorray
Xavier Leon
1
Mauricio R. Alvira
Alberto Auricchio
Stephanie Bucher
Abdelwahed Chtarto
K. Reed Clark
Christophe Darmon
2, 3
Monica Doria
Will Fountain
Guangping Gao
Kai Gao
Mauro Giacca
Juergen Kleinschmidt
Barbara Leuchs
Catherine Melas
Hiroaki Mizukami
Marcus Müller
Yvet Noordman
Olivier Bockstael
Keiya Ozawa
Catherine Pythoud
Marina Sumaroka
Richard Surosky
Liliane Tenenbaum
Inge van der Linden
Brigitte Weins
J. Fraser Wright
Xinhua Zhang
Lorena Zentilin
Fatima Bosch
1
Richard Snyder
Philippe Moullier
2, 3
1
Department of Biochemistry and Molecular Biology [Bellaterra, Spain] (Spain)
- UAB - Universitat Autònoma de Barcelona (UAB Campus 08193 Bellaterra Barcelona - Spain)
- School of Veterinary Medicine [Bellaterra, Spain] (Spain)
2
Inserm UMR 1089 - Laboratoire de Thérapie Génique Translationnelle des Maladies Génétiques (IRS2 Nantes Biotech
22 Boulevard Benoni GOULLIN
44200 Nantes FRANCE - France)
- INSERM - Institut National de la Santé et de la Recherche Médicale (101, rue de Tolbiac, 75013 Paris - France)
- UN - Université de Nantes : UMR1089 (1, quai de Tourville - BP 13522 - 44035 Nantes cedex 1 - France)
3
AGT - Atlantic Gene Therapies [Nantes] (Ile de Nantes - 22 bld Benoni Goullin 44100 Nantes. - France)
- IRS2 Nantes Biotech - Institut de Recherche en Santé 2 – Nantes Biotech (Ile de Nantes - 22 bld Benoni Goullin 44100 Nantes. - France)
4
Perelman School of Medicine - Department of Pathology and Laboratory Medicine [Philadelphia, PA, USA] (United States)
- University of Pennsylvania [Philadelphia] (3451 Walnut Street, Philadelphia, PA 19104 | 215-898-5000 - United States)
Abstract : Gene therapy approaches using recombinant adeno-associated virus serotype 2 (rAAV2) and serotype 8 (rAAV8) have achieved significant clinical benefits. The generation of rAAV Reference Standard Materials (RSM) is key to providing points of reference for particle titer, vector genome titer, and infectious titer for gene transfer vectors. Following the example of the rAAV2RSM, here we have generated and characterized a novel RSM based on rAAV serotype 8. The rAAV8RSM was produced using transient transfection, and the purification was based on density gradient ultracentrifugation. The rAAV8RSM was distributed for characterization along with standard assay protocols to 16 laboratories worldwide. Mean titers and 95% confidence intervals
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Journal articles
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https://www.hal.inserm.fr/inserm-01847281
Contributor : Nicolas Jaulin <>
Submitted on : Monday, August 20, 2018 - 4:24:51 PM
Last modification on : Monday, November 16, 2020 - 4:26:03 PM
Contributor : Nicolas Jaulin <>
Submitted on : Monday, August 20, 2018 - 4:24:51 PM
Last modification on : Monday, November 16, 2020 - 4:26:03 PM
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- HAL Id : inserm-01847281, version 1
- DOI : 10.1089/hum.2014.057
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Citation
Eduard Ayuso, Veronique Blouin, Martin Lock, Susan Mcgorray, Xavier Leon, et al.. Manufacturing and Characterization of a Recombinant Adeno-Associated Virus Type 8 Reference Standard Material. Human Gene Therapy, Mary Ann Liebert, 2014, 25 (11), pp.977 - 987. ⟨10.1089/hum.2014.057⟩. ⟨inserm-01847281⟩
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