Zobrazeno 1 - 10
of 30
pro vyhledávání: '"Marcelo Simon Sola"'
Autor:
Gerasimos Anagnostopoulos, Omar Motiño, Sijing Li, Vincent Carbonnier, Hui Chen, Valentina Sica, Sylvère Durand, Mélanie Bourgin, Fanny Aprahamian, Nitharsshini Nirmalathasan, Romain Donne, Chantal Desdouets, Marcelo Simon Sola, Konstantina Kotta, Léa Montégut, Flavia Lambertucci, Didier Surdez, Grossetête Sandrine, Olivier Delattre, Maria Chiara Maiuri, José Manuel Bravo-SanPedro, Isabelle Martins, Guido Kroemer
Publikováno v:
Cell Death and Disease, Vol 13, Iss 4, Pp 1-15 (2022)
Abstract Acyl-coenzyme-A-binding protein (ACBP), also known as a diazepam-binding inhibitor (DBI), is a potent stimulator of appetite and lipogenesis. Bioinformatic analyses combined with systematic screens revealed that peroxisome proliferator-activ
Externí odkaz:
https://doaj.org/article/9d621ffca98c4f8181d6d34f52f24bcd
Autor:
Giulia De Sabbata, Florence Boisgerault, Corrado Guarnaccia, Alessandra Iaconcig, Giulia Bortolussi, Fanny Collaud, Giuseppe Ronzitti, Marcelo Simon Sola, Patrice Vidal, Jeremy Rouillon, Severine Charles, Emanuele Nicastro, Lorenzo D’Antiga, Petr Ilyinskii, Federico Mingozzi, Takashi Kei Kishimoto, Andrés F. Muro
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 20, Iss , Pp 169-180 (2021)
Ornithine transcarbamylase deficiency (OTCD) is an X-linked liver disorder caused by partial or total loss of OTC enzyme activity. It is characterized by elevated plasma ammonia, leading to neurological impairments, coma, and death in the most severe
Externí odkaz:
https://doaj.org/article/3a96d0d695e64c20a8383d4f8962ab9a
Autor:
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
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 12, Iss , Pp 85-101 (2019)
Hepatocyte-restricted, AAV-mediated gene transfer is being used to provide sustained, tolerogenic transgene expression in gene therapy. However, given the episomal status of the AAV genome, this approach cannot be applied to pediatric disorders when
Externí odkaz:
https://doaj.org/article/a3d927f9d1284167b0026e3289c81292
Autor:
Fanny Collaud, Giulia Bortolussi, Laurence Guianvarc’h, Sem J. Aronson, Thierry Bordet, Philippe Veron, Severine Charles, Patrice Vidal, Marcelo Simon Sola, Stephanie Rundwasser, Delphine G. Dufour, Florence Lacoste, Cyril Luc, Laetitia v. Wittenberghe, Samia Martin, Christine Le Bec, Piter J. Bosma, Andres F. Muro, Giuseppe Ronzitti, Matthias Hebben, Federico Mingozzi
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 12, Iss , Pp 157-174 (2019)
Adeno-associated viruses (AAVs) are among the most efficient vectors for liver gene therapy. Results obtained in the first hemophilia clinical trials demonstrated the long-term efficacy of this approach in humans, showing efficient targeting of hepat
Externí odkaz:
https://doaj.org/article/721441a735dc4663ac629a4a43fbb03b
Autor:
Gerasimos Anagnostopoulos, Omar Motiño, Sijing Li, Vincent Carbonnier, Hui Chen, Valentina Sica, Sylvère Durand, Mélanie Bourgin, Fanny Aprahamian, Nitharsshini Nirmalathasan, Romain Donne, Chantal Desdouets, Marcelo Simon Sola, Konstantina Kotta, Léa Montégut, Flavia Lambertucci, Didier Surdez, Grossetête Sandrine, Olivier Delattre, Maria Chiara Maiuri, José Manuel Bravo-San Pedro, Isabelle Martins, Guido Kroemer
Publikováno v:
Cell Death and Disease, Vol 13, Iss 5, Pp 1-1 (2022)
Externí odkaz:
https://doaj.org/article/079b77792a314dfb93dbcdc0d5aaa91b
Autor:
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
Publikováno v:
Nature Communications, Vol 9, Iss 1, Pp 1-13 (2018)
Immunogenicity of AAV vectors renders repeated AAV dosing ineffective. Here the authors show that coadministration of nanoparticle-encapsulated rapamycin overcomes AAV immunogenicity through Treg induction, enabling efficient AAV redosing in mice and
Externí odkaz:
https://doaj.org/article/8c201057906f4f2d8d990ffb8e12cbae
Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction
Autor:
Zachary Fitzpatrick, Christian Leborgne, Elena Barbon, Elisa Masat, Giuseppe Ronzitti, Laetitia van Wittenberghe, Alban Vignaud, Fanny Collaud, Séverine Charles, Marcelo Simon Sola, Fabienne Jouen, Olivier Boyer, Federico Mingozzi
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 9, Iss , Pp 119-129 (2018)
Pre-existing immunity to adeno-associated virus (AAV) is highly prevalent in humans and can profoundly impact transduction efficiency. Despite the relevance to AAV-mediated gene transfer, relatively little is known about the fate of AAV vectors in th
Externí odkaz:
https://doaj.org/article/c07dc0742e12415387c9f3ce99b267af
Autor:
Amine Meliani, Florence Boisgerault, Zachary Fitzpatrick, Solenne Marmier, Christian Leborgne, Fanny Collaud, Marcelo Simon Sola, Severine Charles, Giuseppe Ronzitti, Alban Vignaud, Laetitia van Wittenberghe, Beatrice Marolleau, Fabienne Jouen, Sisareuth Tan, Olivier Boyer, Olivier Christophe, Alain R. Brisson, Casey A. Maguire, Federico Mingozzi
Publikováno v:
Blood Advances, Vol 1, Iss 23, Pp 2019-2031 (2017)
Abstract: Results from clinical trials of liver gene transfer for hemophilia demonstrate the potential of the adeno-associated virus (AAV) vector platform. However, to achieve therapeutic transgene expression, in some cases high vector doses are requ
Externí odkaz:
https://doaj.org/article/302c51191d2d4ae38857d80e959d437b
Autor:
Giuseppe Ronzitti, Giulia Bortolussi, Remco van Dijk, Fanny Collaud, Severine Charles, Christian Leborgne, Patrice Vidal, Samia Martin, Bernard Gjata, Marcelo Simon Sola, Laetitia van Wittenberghe, Alban Vignaud, Philippe Veron, Piter J Bosma, Andres F Muro, Federico Mingozzi
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 3, Iss C (2016)
Crigler-Najjar syndrome is a severe metabolic disease of the liver due to a reduced activity of the UDP Glucuronosyltransferase 1A1 (UGT1A1) enzyme. In an effort to translate to the clinic an adeno-associated virus vector mediated liver gene transfer
Externí odkaz:
https://doaj.org/article/b411908592d443658eb6cd43556b216e
Autor:
Clément Pontoizeau, Clovis Gaborit, Nolan Tual, Marcelo Simon‐Sola, Irina Rotaru, Marion Benoist, Pasqualina Colella, Antonin Lamazière, Anaïs Brassier, Jean‐Baptiste Arnoux, Agnès Rötig, Chris Ottolenghi, Pascale de Lonlay, Federico Mingozzi, Marina Cavazzana, Manuel Schiff
Publikováno v:
Journal of Inherited Metabolic Disease.