High incidence of severe cyclosporine neurotoxicity in children affected by haemoglobinopaties undergoing myeloablative haematopoietic stem cell transplantation: early diagnosis and prompt intervention ameliorates neurological outcome

Autor: Minicucci Fabio, Vezzulli Paolo, Baldoli Cristina, Finizio Valentina, Biral Erika, Frugnoli Ilaria, Chiesa Robert, Biffi Alessandra, Cappelli Barbara, Noè Anna, Fanelli Giovanna, Fiori Rossana, Ciceri Fabio, Roncarolo Maria, Marktel Sarah

Publikováno v: Italian Journal of Pediatrics, Vol 36, Iss 1, p 14 (2010)

Abstract Background Neurotoxicity is a recognized complication of cyclosporine A (CSA) treatment. The incidence of severe CSA-related neurological complications following hematopoietic stem cell transplantation (HSCT) is 4-11%. Methods We describe 6

Externí odkaz: https://doaj.org/article/f99113ffee2644bc9f64056a5e46d563

Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial

Autor: Sessa, Maria, Lorioli, Laura, Fumagalli, Francesca, Acquati, Serena, Redaelli, Daniela, Baldoli, Cristina, Canale, Sabrina, Lopez, Ignazio D, Morena, Francesco, Calabria, Andrea, Fiori, Rossana, Silvani, Paolo, Rancoita, Paola M V, Gabaldo, Michela, Benedicenti, Fabrizio, Antonioli, Gigliola, Assanelli, Andrea, Cicalese, Maria Pia, del Carro, Ubaldo, Sora, Maria Grazia Natali, Martino, Sabata, Quattrini, Angelo, Montini, Eugenio, Di Serio, Clelia, Ciceri, Fabio, Roncarolo, Maria Grazia, Aiuti, Alessandro, Naldini, Luigi, Biffi, Alessandra ^*

Publikováno v: In The Lancet 30 July-5 August 2016 388(10043):476-487

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Intra-arterial transplantation of HLA-matched donor mesoangioblasts in Duchenne muscular dystrophy

Autor: Cossu, Giulio, Previtali, Stefano C, Napolitano, Sara, Cicalese, Maria Pia, Tedesco, Francesco Saverio, Nicastro, Francesca, Noviello, Maddalena, Roostalu, Urmas, Natali Sora, Maria Grazia, Scarlato, Marina, De Pellegrin, Maurizio, Godi, Claudia, Giuliani, Serena, Ciotti, Francesca, Tonlorenzi, Rossana, Lorenzetti, Isabella, Rivellini, Cristina, Benedetti, Sara, Gatti, Roberto, Marktel, Sarah, Mazzi, Benedetta, Tettamanti, Andrea, Ragazzi, Martina, Imro, Maria Adele, Marano, Giuseppina, Ambrosi, Alessandro, Fiori, Rossana, Sormani, Maria Pia, Bonini, Chiara, Venturini, Massimo, Politi, Letterio S, Torrente, Yvan, Ciceri, Fabio

Publikováno v: EMBO Molecular Medicine
Cossu, G, Previtali, S C, Napolitano, S, Cicalese, M P, Tedesco, F S, Nicastro, F, Noviello, M, Roostalu, U, Natali Sora, M G, Scarlato, M, De Pellegrin, M, Godi, C, Giuliani, S, Ciotti, F, Tonlorenzi, R, Lorenzetti, I, Rivellini, C, Benedetti, S, Gatti, R, Marktel, S, Mazzi, B, Tettamanti, A, Ragazzi, M, Imro, M A, Marano, G, Ambrosi, A, Fiori, R, Sormani, M P, Bonini, C, Venturini, M, Politi, L S, Torrente, Y & Ciceri, F 2015, ' Intra-arterial transplantation of HLA-matched donor mesoangioblasts in Duchenne muscular dystrophy. ', EMBO Molecular Medicine . https://doi.org/10.15252/emmm.201505636
Cossu, G, Previtali, S C, Napolitano, S, Cicalese, M P, Tedesco, F S, Nicastro, F, Noviello, M, Roostalu, U, Natali Sora, M G, Scarlato, M, De Pellegrin, M, Godi, C, Giuliani, S, Ciotti, F, Tonlorenzi, R, Lorenzetti, I, Rivellini, C, Benedetti, S, Gatti, R, Marktel, S, Mazzi, B, Tettamanti, A, Ragazzi, M, Imro, M A, Marano, G, Ambrosi, A, Fiori, R, Sormani, M P, Bonini, C, Venturini, M, Politi, L S, Torrente, Y & Ciceri, F 2015, ' Intra-arterial transplantation of HLA-matched donor mesoangioblasts in Duchenne muscular dystrophy. ', EMBO Molecular Medicine, vol. 7, no. 12, pp. 1513-1528 . https://doi.org/10.15252/emmm.201505636

Intra-arterial transplantation of mesoangioblasts proved safe and partially efficacious in preclinical models of muscular dystrophy. We now report the first-in-human, exploratory, non-randomized open-label phase I-IIa clinical trial of intra-arterial

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d4960b72080285c388f8ebbb8ce13aa3
http://hdl.handle.net/11567/847489

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