Recommendations for pre-symptomatic genetic testing for hereditary transthyretin amyloidosis in the era of effective therapy: a multicenter Italian consensus
Grandis, M, Obici, L, Luigetti, M, Briani, C, Benedicenti, F, Bisogni, G, Canepa, M, Cappelli, F, Danesino, C, Fabrizi, G M, Fenu, S, Ferrandes, G, Gemelli, C, Manganelli, F, Mazzeo, A, Melchiorri, L, Perfetto, F, Pradotto, L G, Rimessi, P, Tini, G, Tozza, S, Trevisan, L, Pareyson, D, Mandich, P
Published in Orphanet journal of rare diseases (14.12.2020)
Published in Orphanet journal of rare diseases (14.12.2020)
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Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy
Lattanzi, Annalisa, Salvagno, Camilla, Maderna, Claudio, Benedicenti, Fabrizio, Morena, Francesco, Kulik, Willem, Naldini, Luigi, Montini, Eugenio, Martino, Sabata, Gritti, Angela
Published in Human molecular genetics (15.06.2014)
Published in Human molecular genetics (15.06.2014)
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Early onset developmental and epileptic encephalopathy and Rett-like phenotype in a 15-year-old girl affected by Cornelia de Lange syndrome type 2 due to a SMC1A gene mutation
Parmeggiani, L, Stanzial, F, Menna, E, Boni, E, Manzoni, F, Benedicenti, F, Pellegrin, S
Published in Epilepsy & behavior reports (01.01.2023)
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Shedding of clinical-grade lentiviral vectors is not detected in a gene therapy setting
Cesani, M, Plati, T, Lorioli, L, Benedicenti, F, Redaelli, D, Dionisio, F, Biasco, L, Montini, E, Naldini, L, Biffi, A
Published in Gene therapy (01.06.2015)
Published in Gene therapy (01.06.2015)
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Preclinical evaluation of donor-derived sleeping beauty modified CD19CAR+ lymphocytes for the treatment of acute lymphoblastic leukemia
Magnani, C, Mezzanotte, C, Cappuzzello, C, Benedicenti, F, Belotti, D, Cabiati, B, Bardini, M, Fazio, G, Cazzaniga, G, Cooper, L, Montini, E, Gaipa, G, Biondi, A, Biagi, E
Published in Cytotherapy (Oxford, England) (01.05.2017)
Published in Cytotherapy (Oxford, England) (01.05.2017)
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An intronic mutation causes severe LGMD2A in a large inbred family belonging to a genetic isolate in the Alps
Fanin, M, Benedicenti, F, Fritegotto, C, Nascimbeni, AC, Peterle, E, Stanzial, F, Cristofoletti, A, Castellan, C, Angelini, C
Published in Clinical genetics (01.12.2012)
Published in Clinical genetics (01.12.2012)
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Cytokine-induced killer (CIK) cells engineered with chimeric antigen receptors (CARs) by sleeping beauty system
Magnani, C, Turazzi, N, Benedicenti, F, Tettamanti, S, Attianese, G. Giordano, Rossi, V, Montini, E, Cooper, L, Aiuti, A, Biondi, A, Biagi, E
Published in Cytotherapy (Oxford, England) (01.04.2014)
Published in Cytotherapy (Oxford, England) (01.04.2014)
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Novel human pathological mutations. Gene symbol: HMBS. Disease: Porphyria, acute intermittent
Di Pierro, Elena, Brancaleoni, V, Stanzial, F, Benedicenti, F, Castellan, C, Cappellini, M D
Published in Human genetics (01.08.2009)
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Published in Human genetics (01.08.2009)
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OC.02.3: EFFICACY OF A DESIGNED KIT FOR THE DIAGNOSIS OF NONCELIAC GLUTEN SENSITIVITYAND GLUTEN-RELATED SYMPTOMS
Elli, L., Bascuñán, K.A., Costantino, A., Benedicenti, F., Doneda, L., Scricciolo, A., Lombardo, V., Roncoroni, L.
Published in Digestive and liver disease (01.04.2024)
Published in Digestive and liver disease (01.04.2024)
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OC.01.6: COLD SNARE POLYPECTOMY IN THE MANAGEMENT OF DUODENAL ADENOMA IN FAMILIAL ADENOMATOUS POLYPOSIS
Cavalcoli, F., Magarotto, A., Emanuele, R., Rosa, R., Borsotti, E., Mancini, A., Benedicenti, F., Lauricella, S., Vitellaro, M., Cantù, P.
Published in Digestive and liver disease (01.04.2024)
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Liver-directed lentiviral gene therapy in a dog model of hemophilia B
Cantore, Alessio, Ranzani, Marco, Bartholomae, Cynthia C, Volpin, Monica, Valle, Patrizia Della, Sanvito, Francesca, Sergi, Lucia Sergi, Gallina, Pierangela, Benedicenti, Fabrizio, Bellinger, Dwight, Raymer, Robin, Merricks, Elizabeth, Bellintani, Francesca, Martin, Samia, Doglioni, Claudio, D'Angelo, Armando, VandenDriessche, Thierry, Chuah, Marinee K, Schmidt, Manfred, Nichols, Timothy, Montini, Eugenio, Naldini, Luigi
Published in Science translational medicine (04.03.2015)
Published in Science translational medicine (04.03.2015)
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Dietary Fiber and Fructooligosaccharides (FOS) intake in adult patients with inflammatory bowel
Scricciolo, A., Bascunan, K., Araya, M., Lombardo, V., Costantino, A., Benedicenti, F., Elli, L., Caprioli, F., Tontini, G.E., Vecchi, M., Doneda, L., Roncoroni, L.
Published in Clinical nutrition ESPEN (01.10.2024)
Published in Clinical nutrition ESPEN (01.10.2024)
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28 - Preclinical evaluation of donor-derived sleeping beauty modified CD19CAR+ lymphocytes for the treatment of acute lymphoblastic leukemia
Magnani, C., Mezzanotte, C., Cappuzzello, C., Benedicenti, F., Belotti, D., Cabiati, B., Bardini, M., Fazio, G., Cazzaniga, G., Cooper, L., Montini, E., Gaipa, G., Biondi, A., Biagi, E.
Published in Cytotherapy (Oxford, England) (01.05.2017)
Published in Cytotherapy (Oxford, England) (01.05.2017)
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Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells
De Palma, Michele, Montini, Eugenio, de Sio, Francesca R.Santoni, Benedicenti, Fabrizio, Gentile, Alessandra, Medico, Enzo, Naldini, Luigi
Published in Blood (15.03.2005)
Published in Blood (15.03.2005)
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MKS3/TMEM67 mutations are a major cause of COACH Syndrome, a Joubert Syndrome related disorder with liver involvement
Brancati, Francesco, Iannicelli, Miriam, Travaglini, Lorena, Mazzotta, Annalisa, Bertini, Enrico, Boltshauser, Eugen, D'Arrigo, Stefano, Emma, Francesco, Fazzi, Elisa, Gallizzi, Romina, Gentile, Mattia, Loncarevic, Damir, Mejaski-Bosnjak, Vlatka, Pantaleoni, Chiara, Rigoli, Luciana, Salpietro, Carmelo D, Signorini, Sabrina, Stringini, Gilda Rita, Verloes, Alain, Zabloka, Dominika, Dallapiccola, Bruno, Gleeson, Joseph G, Valente, Enza Maria
Published in Human mutation (01.02.2009)
Published in Human mutation (01.02.2009)
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Prenatal Detection of Congenital Renal Malformations by Fetal Ultrasonographic Examination: An Analysis of 709,030 Births in 12 European Countries
Wiesel, A., Queisser-Luft, A., Clementi, M., Bianca, S., Stoll, C.
Published in European journal of medical genetics (01.04.2005)
Published in European journal of medical genetics (01.04.2005)
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Expanding CEP290 mutational spectrum in ciliopathies
Travaglini, Lorena, Brancati, Francesco, Attie-Bitach, Tania, Audollent, Sophie, Bertini, Enrico, Kaplan, Josseline, Perrault, Isabelle, Iannicelli, Miriam, Mancuso, Brunella, Rigoli, Luciana, Rozet, Jean-Michel, Swistun, Dominika, Tolentino, Jerlyn, Dallapiccola, Bruno, Gleeson, Joseph G., Valente, Enza Maria
Published in American journal of medical genetics. Part A (01.10.2009)
Published in American journal of medical genetics. Part A (01.10.2009)
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