Zobrazeno 1 - 10
of 117
pro vyhledávání: '"A F, Muro"'
Publikováno v:
Frontiers in Genome Editing, Vol 6 (2024)
The liver is an essential organ of the body that performs several vital functions, including the metabolism of biomolecules, foreign substances, and toxins, and the production of plasma proteins, such as coagulation factors. There are hundreds of gen
Externí odkaz:
https://doaj.org/article/32109d96a87b414185b62162a0b6742f
Autor:
Héctor Franco-Valls, Elsa Tusquets-Uxó, Laura Sala, Maria Val, Raúl Peña, Alessandra Iaconcig, Álvaro Villarino, Martín Jiménez-Arriola, Pere Massó, Juan L. Trincado, Eduardo Eyras, Andrés F. Muro, Jorge Otero, Antonio García de Herreros, Josep Baulida
Publikováno v:
Breast Cancer Research, Vol 25, Iss 1, Pp 1-19 (2023)
Abstract Background As in most solid cancers, the emergence of cells with oncogenic mutations in the mammary epithelium alters the tissue homeostasis. Some soluble factors, such as TGFβ, potently modify the behavior of healthy stromal cells. A subpo
Externí odkaz:
https://doaj.org/article/8319198f0e46428e87b3865f27609b45
Autor:
Michela Lisjak, Alessandra Iaconcig, Corrado Guarnaccia, Antonio Vicidomini, Laura Moretti, Fanny Collaud, Giuseppe Ronzitti, Lorena Zentilin, Andrés F. Muro
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 31, Iss , Pp 101103- (2023)
Citrullinemia type I is a rare autosomal-recessive disorder caused by deficiency of argininosuccinate synthetase (ASS1). The clinical presentation includes the acute neonatal form, characterized by ammonia and citrulline accumulation in blood, which
Externí odkaz:
https://doaj.org/article/a42a41ea318440d88db0d7c7e952dfc5
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:
Xiaoxia Shi, Sem J. Aronson, Lysbeth ten Bloemendaal, Suzanne Duijst, Robert S. Bakker, Dirk R. de Waart, Giulia Bortolussi, Fanny Collaud, Ronald P. Oude Elferink, Andrés F. Muro, Federico Mingozzi, Giuseppe Ronzitti, Piter J. Bosma
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 20, Iss , Pp 287-297 (2021)
A clinical trial using adeno-associated virus serotype 8 (AAV8)-human uridine diphosphate glucuronosyltransferase 1A1 (hUGT1A1) to treat inherited severe unconjugated hyperbilirubinemia (Crigler-Najjar syndrome) is ongoing, but preclinical data sugge
Externí odkaz:
https://doaj.org/article/5f312cc89674405188672f6d384e4ef1
Autor:
Leandro R Soria, Sonam Gurung, Giulia De Sabbata, Dany P Perocheau, Angela De Angelis, Gemma Bruno, Elena Polishchuk, Debora Paris, Paola Cuomo, Andrea Motta, Michael Orford, Youssef Khalil, Simon Eaton, Philippa B Mills, Simon N Waddington, Carmine Settembre, Andrés F Muro, Julien Baruteau, Nicola Brunetti‐Pierri
Publikováno v:
EMBO Molecular Medicine, Vol 13, Iss 2, Pp 1-14 (2020)
Abstract Urea cycle disorders (UCD) are inherited defects in clearance of waste nitrogen with high morbidity and mortality. Novel and more effective therapies for UCD are needed. Studies in mice with constitutive activation of autophagy unravelled Be
Externí odkaz:
https://doaj.org/article/19085dda865a4f648e70bb866bfbf0bc
Autor:
Sem J. Aronson, Robert S. Bakker, Sascha Moenis, Remco van Dijk, Giulia Bortolussi, Fanny Collaud, Xiaoxia Shi, Suzanne Duijst, Lysbeth ten Bloemendaal, Giuseppe Ronzitti, Andrés F. Muro, Federico Mingozzi, Ulrich Beuers, Piter J. Bosma
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 18, Iss , Pp 250-258 (2020)
Potency assessment of clinical-grade vector lots is crucial to support adeno-associated virus (AAV) vector release and is required for future marketing authorization. We have developed and validated a cell-based, quantitative potency assay that detec
Externí odkaz:
https://doaj.org/article/edb7a29586e044c8b082be4c5aa3a2dd
Autor:
Michela Lisjak, Alessia De Caneva, Thibaut Marais, Elena Barbon, Maria Grazia Biferi, Fabiola Porro, Adi Barzel, Lorena Zentilin, Mark A. Kay, Federico Mingozzi, Andrés F. Muro
Publikováno v:
Frontiers in Genome Editing, Vol 4 (2022)
Many inborn errors of metabolism require life-long treatments and, in severe conditions involving the liver, organ transplantation remains the only curative treatment. Non-integrative AAV-mediated gene therapy has shown efficacy in adult patients. Ho
Externí odkaz:
https://doaj.org/article/8b673b0e33fa4dbfbfb0304e87ef4951
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:
Xiaoxia Shi, Giulia Bortolussi, Lysbeth Ten Bloemendaal, Suzanne Duijst, Andrés F Muro, Piter J Bosma
Publikováno v:
PLoS ONE, Vol 16, Iss 4, p e0250605 (2021)
In contrast to AAV, Simian Virus 40 (rSV40) not inducing neutralizing antibodies (NAbs) allowing re-treatment seems a promising vector for neonatal treatment of inherited liver disorders. Several studies have reported efficacy of rSV40 in animal mode
Externí odkaz:
https://doaj.org/article/a08eb17d84844a7a8088f691c132c137