| Rescue of bilirubin-induced neonatal lethality in a mouse model of Crigler-Najjar syndrome type I by AAV9-mediated gene transfer G Bortolussi, L Zentilin, G Baj, P Giraudi, C Bellarosa, M Giacca, C Tiribelli, ... The FASEB Journal 26 (3), 1052-1063, 2012 | 90 | 2012 |
| Life-long correction of hyperbilirubinemia with a neonatal liver-specific AAV-mediated gene transfer in a lethal mouse model of Crigler–Najjar Syndrome G Bortolussi, L Zentillin, J Vaníkova, L Bockor, C Bellarosa, A Mancarella, ... Human gene therapy 25 (9), 844-855, 2014 | 89 | 2014 |
| Promoterless gene targeting without nucleases rescues lethality of a Crigler‐Najjar syndrome mouse model F Porro, G Bortolussi, A Barzel, A De Caneva, A Iaconcig, S Vodret, ... EMBO molecular medicine 9 (10), 1346-1355, 2017 | 70 | 2017 |
| A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome G Ronzitti, G Bortolussi, R Van Dijk, F Collaud, S Charles, C Leborgne, ... Molecular Therapy-Methods & Clinical Development 3, 16049, 2016 | 65 | 2016 |
| The role of ABC transporters in protecting cells from bilirubin toxicity C Bellarosa, G Bortolussi, C Tiribelli Current pharmaceutical design 15 (25), 2884-2892, 2009 | 56 | 2009 |
| Bilirubin-induced oxidative stress leads to DNA damage in the cerebellum of hyperbilirubinemic neonatal mice and activates DNA double-strand break repair pathways in human cells V Rawat, G Bortolussi, S Gazzin, C Tiribelli, AF Muro Oxidative Medicine and Cellular Longevity 2018, 2018 | 55 | 2018 |
| Preclinical development of an AAV8-hUGT1A1 vector for the treatment of Crigler-Najjar syndrome F Collaud, G Bortolussi, L Guianvarc’h, SJ Aronson, T Bordet, P Veron, ... Molecular Therapy-Methods & Clinical Development 12, 157-174, 2019 | 54 | 2019 |
| Age-dependent pattern of cerebellar susceptibility to bilirubin neurotoxicity in vivo in mice G Bortolussi, G Baj, S Vodret, G Viviani, T Bittolo, AF Muro Disease models & mechanisms 7 (9), 1057-1068, 2014 | 49 | 2014 |
| Attenuation of neuro-inflammation improves survival and neurodegeneration in a mouse model of severe neonatal hyperbilirubinemia S Vodret, G Bortolussi, A Iaconcig, E Martinelli, C Tiribelli, AF Muro Brain, behavior, and immunity 70, 166-178, 2018 | 46 | 2018 |
| Coupling AAV-mediated promoterless gene targeting to SaCas9 nuclease to efficiently correct liver metabolic diseases A De Caneva, F Porro, G Bortolussi, R Sola, M Lisjak, A Barzel, M Giacca, ... JCI insight, 2019 | 45 | 2019 |
| Inflammatory signature of cerebellar neurodegeneration during neonatal hyperbilirubinemia in Ugt1-/-mouse model S Vodret, G Bortolussi, J Jašprová, L Vitek, AF Muro Journal of neuroinflammation 14 (1), 64, 2017 | 41 | 2017 |
| Impairment of enzymatic antioxidant defenses is associated with bilirubin-induced neuronal cell death in the cerebellum of Ugt1 KO mice G Bortolussi, E Codarin, G Antoniali, C Vascotto, S Vodret, S Arena, ... Cell death & disease 6 (5), e1739, 2015 | 37 | 2015 |
| Albumin administration prevents neurological damage and death in a mouse model of severe neonatal hyperbilirubinemia S Vodret, G Bortolussi, AB Schreuder, J Jašprová, L Vitek, HJ Verkade, ... Scientific reports 5, 16203, 2015 | 33 | 2015 |
| Repeated AAV-mediated gene transfer by serotype switching enables long-lasting therapeutic levels of hUgt1a1 enzyme in a mouse model of Crigler–Najjar Syndrome Type I L Bočkor, G Bortolussi, A Iaconcig, G Chiaruttini, C Tiribelli, M Giacca, ... Gene therapy 24 (10), 649, 2017 | 32 | 2017 |
| Modulation of bilirubin neurotoxicity by the Abcb1 transporter in the Ugt1-/- lethal mouse model of neonatal hyperbilirubinemia L Bočkor, G Bortolussi, S Vodret, A Iaconcig, J Jašprová, J Zelenka, ... Human molecular genetics 26 (1), 145-157, 2016 | 24 | 2016 |
| Advances in understanding disease mechanisms and potential treatments for Crigler–Najjar syndrome G Bortolussi, AF Muro Expert opinion on orphan drugs 6 (7), 425-439, 2018 | 23 | 2018 |
| Treatment of hyperbilirubinemia F Mingozzi, G Ronzitti, F Collaud, A Muro, G Bortolussi US Patent App. 15/303,834, 2017 | 17 | 2017 |
| Generation of Ugt1-deficient murine liver cell lines using TALEN technology F Porro, L Bockor, A De Caneva, G Bortolussi, AF Muro PloS one 9 (8), e104816, 2014 | 15 | 2014 |
| Serum Bilirubin Levels and Promoter Variations in HMOX1 and UGT1A1 Genes in Patients with Fabry Disease A Jirásková, G Bortolussi, G Dostálová, L Eremiášová, L Golaň, V Danzig, ... Oxidative medicine and cellular longevity 2017, 2017 | 5 | 2017 |
| ESGCT XXV Anniversary Congress in Collaboration with the German Society for Gene Therapy October 17–20, 2017 Berlin, Germany SM Maffioletti, S Sarcar, A Henderson, I Mannhardt, L Pinton, LA Moyle, ... Human Gene Therapy 28 (12), A1-A125, 2017 | 2 | 2017 |
