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

Project Information

MYOCURE

Grant agreement ID: 667751

DOI 10.3030/667751

Project closed

EC signature date 1 December 2015

Start date 1 January 2016

End date 31 December 2019

Funded under

• SOCIETAL CHALLENGES - Health, demographic change and well-being

Total cost € 5 998 937,50

EU contribution € 5 998 937,50

Coordinated by

VRIJE UNIVERSITEIT BRUSSEL
Belgium

Deliverables

Websites, patent fillings, videos etc. (1)

Project website

Publications

Peer reviewed articles (11)

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

Author(s): Dirk Grimm, Hildegard Büning
Published in: Human Gene Therapy, Issue 28/11, 2017, Page(s) 1075-1086, ISSN 1043-0342
Publisher: 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

Author(s): 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
Published in: Science Translational Medicine, Issue 9/418, 2017, Page(s) eaam6375, ISSN 1946-6234
Publisher: 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

Author(s): 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
Published in: Molecular Therapy - Methods & Clinical Development, Issue 6, 2017, Page(s) 1-7, ISSN 2329-0501
Publisher: 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

Author(s): 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
Published in: Nature Communications, Issue 9/1, 2018, ISSN 2041-1723
Publisher: Nature Publishing Group
DOI: 10.1038/s41467-018-06621-3

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

Author(s): Klaudia Kuranda, Priscilla Jean-Alphonse, Christian Leborgne, Romain Hardet, Fanny Collaud, Solenne Marmier, Helena Costa Verdera, Giuseppe Ronzitti, Philippe Veron, Federico Mingozzi
Published in: Journal of Clinical Investigation, Issue 128/12, 2018, Page(s) 5267-5279, ISSN 0021-9738
Publisher: American Society for Clinical Investigation
DOI: 10.1172/jci122372

FGF21 gene therapy as treatment for obesity and insulin resistance

Author(s): 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
Published in: EMBO Molecular Medicine, Issue 10/8, 2018, Page(s) e8791, ISSN 1757-4676
Publisher: 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

Author(s): 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
Published in: Molecular Therapy - Methods & Clinical Development, Issue 12, 2019, Page(s) 85-101, ISSN 2329-0501
Publisher: 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

Author(s): Laia Vil, Carles Roca, Ivet Elias, Alba Casellas, Ricardo Lage, Sylvie Franckhauser, Fatima Bosch
Published in: Molecular Therapy - Methods & Clinical Development, Issue 3, 2016, Page(s) 16072, ISSN 2329-0501
Publisher: 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

Author(s): 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
Published in: Scientific Reports, Issue 6/1, 2016, ISSN 2045-2322
Publisher: Nature Publishing Group
DOI: 10.1038/srep36182

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

Author(s): 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
Published in: Nature Communications, Issue 10/1, 2019, ISSN 2041-1723
Publisher: Nature Publishing Group
DOI: 10.1038/s41467-018-08283-7

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

Author(s): Warut Tulalamba, Jonas Weinmann, Quang Hong Pham, Jihad El Andari, Thierry VandenDriessche, Marinee K. Chuah, Dirk Grimm
Published in: Gene Therapy, 2019, ISSN 0969-7128
Publisher: Nature Publishing Group
DOI: 10.1038/s41434-019-0106-3