Giulia Bortolussi
International Centre for Genetic Engineering and Biotechnology
35 Papers
49 Citations
Giulia Bortolussi is an academic researcher from International Centre for Genetic Engineering and Biotechnology. The author has contributed to research in topics: Kernicterus & Bilirubin. The author has an hindex of 14, co-authored 28 publications. Previous affiliations of Giulia Bortolussi include Charles University in Prague.
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Papers
A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome
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,Andrés F. Muro,Federico Mingozzi,Federico Mingozzi +16 more
TL;DR: The results indicate that transgene codon-optimization is a strategy that can improve efficacy of gene transfer but needs to be carefully tested in vitro and in vivo and support further translation of the approach to humans.
57
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.
TL;DR: It is demonstrated that DNA damage occurs in vivo in the cerebellum, the brain region most affected by bilirubin toxicity, and the mechanisms associated with potential toxic action of bilirUBin on DNA in in vitro models are studied.
Promoterless gene targeting without nucleases rescues lethality of a Crigler-Najjar syndrome mouse model.
Fabiola Porro,Giulia Bortolussi,Adi Barzel,Alessia De Caneva,Alessandra Iaconcig,Simone Vodret,Lorena Zentilin,Mark A. Kay,Andrés F. Muro +8 more
TL;DR: Results proved that the promoterless gene therapy is applicable for CNSI, providing therapeutic levels of an intracellular ER membrane‐bound enzyme responsible for a lethal liver metabolic disease.
56
Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome.
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 van Wittenberghe,Samia Martin,Christine Le Bec,Piter J. Bosma,Andrés F. Muro,Giuseppe Ronzitti,Matthias Hebben,Federico Mingozzi +20 more
TL;DR: An (ss)AAV8 vector carrying the human UDP-glucuronosyltransferase family 1-member A1 (hUGT1A1) transgene under the control of a liver-specific promoter is developed, supporting the clinical translation of this gene therapy approach for the treatment of Crigler-Najjar (CN) syndrome.
51
Coupling AAV-mediated promoterless gene targeting to SaCas9 nuclease to efficiently correct liver metabolic diseases
Alessia De Caneva,Fabiola Porro,Giulia Bortolussi,Riccardo Sola,Michela Lisjak,Adi Barzel,Mauro Giacca,Mark A. Kay,Kristian Vlahoviček,Lorena Zentilin,Andrés F. Muro +10 more
TL;DR: The improved efficacy and reassuring safety profile support the potential application of the proposed AAV-mediated gene therapy in the liver to other liver diseases.