Development of an innovative gene therapy platform to cure rare hereditary muscle disorders

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Small But Increasingly Mighty: Latest Advances in AAV Vector Research, Design, and Evolution

Autores: Dirk Grimm, Hildegard Büning
Publicado en: Human Gene Therapy, Edición 28/11, 2017, Página(s) 1075-1086, ISSN 1043-0342
Editor: Mary Ann Liebert Inc.
DOI: 10.1089/hum.2017.172

Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase

Autores: Francesco Puzzo, Pasqualina Colella, Maria G. Biferi, Deeksha Bali, Nicole K. Paulk, Patrice Vidal, Fanny Collaud, Marcelo Simon-Sola, Severine Charles, Romain Hardet, Christian Leborgne, Amine Meliani, Mathilde Cohen-Tannoudji, Stephanie Astord, Bernard Gjata, Pauline Sellier, Laetitia van Wittenberghe, Alban Vignaud, Florence Boisgerault, Martine Barkats, Pascal Laforet, Mark A. Kay, Dwight D. K
Publicado en: Science Translational Medicine, Edición 9/418, 2017, Página(s) eaam6375, ISSN 1946-6234
Editor: American Association for the Advancement of Science
DOI: 10.1126/scitranslmed.aam6375

Long-Term Efficacy and Safety of Insulin and Glucokinase Gene Therapy for Diabetes: 8-Year Follow-Up in Dogs

Autores: Maria Luisa Jaén, Laia Vilà, Ivet Elias, Veronica Jimenez, Jordi Rodó, Luca Maggioni, Rafael Ruiz-de Gopegui, Miguel Garcia, Sergio Muñoz, David Callejas, Eduard Ayuso, Tura Ferré, Iris Grifoll, Anna Andaluz, Jesus Ruberte, Virginia Haurigot, Fatima Bosch
Publicado en: Molecular Therapy - Methods & Clinical Development, Edición 6, 2017, Página(s) 1-7, ISSN 2329-0501
Editor: Cell Press
DOI: 10.1016/j.omtm.2017.03.008

Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration

Autores: Amine Meliani, Florence Boisgerault, Romain Hardet, Solenne Marmier, Fanny Collaud, Giuseppe Ronzitti, Christian Leborgne, Helena Costa Verdera, Marcelo Simon Sola, Severine Charles, Alban Vignaud, Laetitia van Wittenberghe, Giorgia Manni, Olivier Christophe, Francesca Fallarino, Christopher Roy, Alicia Michaud, Petr Ilyinskii, Takashi Kei Kishimoto, Federico Mingozzi
Publicado en: Nature Communications, Edición 9/1, 2018, ISSN 2041-1723
Editor: Nature Publishing Group
DOI: 10.1038/s41467-018-06621-3

Exposure to wild-type AAV drives distinct capsid immunity profiles in humans

Autores: Klaudia Kuranda, Priscilla Jean-Alphonse, Christian Leborgne, Romain Hardet, Fanny Collaud, Solenne Marmier, Helena Costa Verdera, Giuseppe Ronzitti, Philippe Veron, Federico Mingozzi
Publicado en: Journal of Clinical Investigation, Edición 128/12, 2018, Página(s) 5267-5279, ISSN 0021-9738
Editor: American Society for Clinical Investigation
DOI: 10.1172/jci122372

FGF21 gene therapy as treatment for obesity and insulin resistance

Autores: Veronica Jimenez, Claudia Jambrina, Estefania Casana, Victor Sacristan, Sergio Muñoz, Sara Darriba, Jordi Rodó, Cristina Mallol, Miquel Garcia, Xavier León, Sara Marcó, Albert Ribera, Ivet Elias, Alba Casellas, Ignasi Grass, Gemma Elias, Tura Ferré, Sandra Motas, Sylvie Franckhauser, Francisca Mulero, Marc Navarro, Virginia Haurigot, Jesus Ruberte, Fatima Bosch
Publicado en: EMBO Molecular Medicine, Edición 10/8, 2018, Página(s) e8791, ISSN 1757-4676
Editor: John Wiley & Sons Ltd.
DOI: 10.15252/emmm.201708791

AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice

Autores: Pasqualina Colella, Pauline Sellier, Helena Costa Verdera, Francesco Puzzo, Laetitia van Wittenberghe, Nicolas Guerchet, Nathalie Daniele, Bernard Gjata, Solenne Marmier, Severine Charles, Marcelo Simon Sola, Isabella Ragone, Christian Leborgne, Fanny Collaud, Federico Mingozzi
Publicado en: Molecular Therapy - Methods & Clinical Development, Edición 12, 2019, Página(s) 85-101, ISSN 2329-0501
Editor: Cell Press
DOI: 10.1016/j.omtm.2018.11.002

AAV-mediated Sirt1 overexpression in skeletal muscle activates oxidative capacity but does not prevent insulin resistance

Autores: Laia Vil, Carles Roca, Ivet Elias, Alba Casellas, Ricardo Lage, Sylvie Franckhauser, Fatima Bosch
Publicado en: Molecular Therapy - Methods & Clinical Development, Edición 3, 2016, Página(s) 16072, ISSN 2329-0501
Editor: Elsevier
DOI: 10.1038/mtm.2016.72

Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients

Autores: Elisa Masat, Pascal Laforêt, Marie De Antonio, Guillaume Corre, Barbara Perniconi, Nadjib Taouagh, Kuberaka Mariampillai, Damien Amelin, Wladimir Mauhin, Jean-Yves Hogrel, Catherine Caillaud, Giuseppe Ronzitti, Francesco Puzzo, Klaudia Kuranda, Pasqualina Colella, Roberto Mallone, Olivier Benveniste, Federico Mingozzi, G. Bassez, A. L. Bedat-Millet, A. Behin, B. Eymard, S. Leonard-Louis, T. Stojk
Publicado en: Scientific Reports, Edición 6/1, 2016, ISSN 2045-2322
Editor: Nature Publishing Group
DOI: 10.1038/srep36182

Next-generation muscle-directed gene therapy by in silico vector design

Autores: S. Sarcar, W. Tulalamba, M. Y. Rincon, J. Tipanee, H. Q. Pham, H. Evens, D. Boon, E. Samara-Kuko, M. Keyaerts, M. Loperfido, E. Berardi, S. Jarmin, P. In’t Veld, G. Dickson, T. Lahoutte, M. Sampaolesi, P. De Bleser, T. VandenDriessche, M. K. Chuah
Publicado en: Nature Communications, Edición 10/1, 2019, ISSN 2041-1723
Editor: Nature Publishing Group
DOI: 10.1038/s41467-018-08283-7

Distinct transduction of muscle tissue in mice after systemic delivery of AAVpo1 vectors

Autores: Warut Tulalamba, Jonas Weinmann, Quang Hong Pham, Jihad El Andari, Thierry VandenDriessche, Marinee K. Chuah, Dirk Grimm
Publicado en: Gene Therapy, 2019, ISSN 0969-7128
Editor: Nature Publishing Group
DOI: 10.1038/s41434-019-0106-3

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