Andrés Fernando Muro

Group Leader,
Mouse Molecular Genetics
International Centre for Genetic Engineering and Biotechnology
Padriciano 99
34149 Trieste, Italy
E-mail: [email protected] 
Tel: +39-040-3757369/12


School of Sciences, University of Buenos Aires, Buenos Aires, Argentina, PhD in Biology, 1992
School of Sciences, University of Buenos Aires, Buenos Aires, Argentina, MSc in Molecular Biology, 1988

Career History

Since 2005, Group Leader of the Mouse Molecular Genetics Laboratory of the International Centre of Genetic Engineering and Biotechnology (ICGEB), Trieste, Italy.
1995-2004, Staff Scientist, Molecular Pathology Laboratory, ICGEB, Trieste, Italy.
1992-1994, Postdoc, Molecular Pathology Laboratory, ICGEB, Trieste, Italy.
1991, Visiting scientist, Molecular Pathology Laboratory, ICGEB, Trieste, Italy.
1988-1992, Graduate Student Fellowship, Consejo Nacional de Investigaciones Científicas y Técnicas (CONICET) at the Instituto de Ingenieria Genetica y Biologia Molecular (INGEBI-CONICET), Buenos Aires, Argentina.
1987-1988, Pre-doctoral student at Laboratorio de Biologia Molecular, Facultad de Ciencias Exactas y Naturales, Universidad de Buenos Aires, Argentina.

Scientific Activity

Dr. Muro’s main focus of research is the understanding of gene function and their relation to diseases, and the development of efficient therapeutic approaches, using mouse models, with special emphasis in metabolic liver diseases, in particular in the study of the mechanisms of disease and possible therapies of neonatal hyperbilirubinemia and the Crigler-Najjar syndrome type I. He is interested in the understanding of the mechanisms of bilirubin neurotoxicity, in order to develop pharmacological and gene therapy approaches aimed to avoid severe and permanent brain damage, and lethality.

In parallel to the work in CNSI, he is developing a therapeutic vector to treat patients suffering from ornithine transcarbamylase deficiency.
Dr. Muro is participating in international research and clinical networks aiming to transfer the developed therapies to the clinics.
Research activity is supported through grants from public and private agencies, including the Telethon, Genethon, and the European Commission (H2020), as well as through collaborations with companies (Selecta Biosciences, US).

Selected publications

D’Antiga L,  Beuers U,  Ronzitti G,  Brunetti-Pierri N,  Baumann U,  Di Giorgio A,  Aronson S,  Hubert A, Romano R,  Junge N,  Bosma P,  Bortolussi G,  Muro A. et al. 2023. Gene Therapy in Patients with the Crigler–Najjar Syndrome. N Engl J Med 389:620-631.

Collaud F, Bortolussi G, Guianvarc’h L, Aronson SJ, Bordet T, Veron P, Charles S, Vidal P, Sola MS, Rundwasser S, Dufour DG, Lacoste F, Luc C, Wittenberghe LV, Martin S, Le Bec C, Bosma PJ, Muro AF, Ronzitti G, Hebben M, Mingozzi F. 2019. Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome. Mol Ther Methods Clin Dev 12:157-174.

Bockor L, Bortolussi G, Iaconcig A, Chiaruttini G, Tiribelli C, Giacca M, Benvenuti F, Zentilin L, Muro AF. 2017. 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. Gene Ther 24:649-660.

Vodret S, Bortolussi G, Jasprova J, Vitek L, Muro AF. 2017. Inflammatory signature of cerebellar neurodegeneration during neonatal hyperbilirubinemia in Ugt1 -/- mouse model. J Neuroinflammation 14:64.

Bortolussi G, Zentillin L, Vanikova J, Bockor L, Bellarosa C, Mancarella A, Vianello E, Tiribelli C, Giacca M, Vitek L, Muro AF. 2014. Life-long correction of hyperbilirubinemia with a neonatal liver-specific AAV-mediated gene transfer in a lethal mouse model of Crigler-Najjar Syndrome. Hum Gene Ther 25:844-855.

Bortolussi G, Zentilin L, Baj G, Giraudi P, Bellarosa C, Giacca M, Tiribelli C, Muro AF. 2012. Rescue of bilirubin-induced neonatal lethality in a mouse model of Crigler-Najjar syndrome type I by AAV9-mediated gene transfer. FASEB J 26:1052-1063.

Morgan M, Iaconcig A, Muro AF. 2010. CPEB2, CPEB3 and CPEB4 are coordinately regulated by miRNAs recognizing conserved binding sites in paralog positions of their 3′-UTRs. Nucleic Acids Res 38:7698-7710.

Bazigou E, Xie S, Chen C, Weston A, Miura N, Sorokin L, Adams R, Muro AF, Sheppard D, Makinen T. 2009. Integrin-alpha9 is required for fibronectin matrix assembly during lymphatic valve morphogenesis. Dev Cell 17:175-186.

Muro AF, Chauhan AK, Gajovic S, Iaconcig A, Porro F, Stanta G, Baralle FE. 2003. Regulated splicing of the fibronectin EDA exon is essential for proper skin wound healing and normal lifespan. J Cell Biol 162:149-160.

Muro AF, Marro ML, Gajovic S, Porro F, Luzzatto L, Baralle FE. 2000. Mild spherocytic hereditary elliptocytosis and altered levels of alpha- and gamma-adducins in beta-adducin-deficient mice. Blood 95:3978-3985.

Muro AF, Moretti FA, Moore BB, Yan M, Atrasz RG, Wilke CA, Flaherty KR, Martinez FJ, Tsui JL, Sheppard D, Baralle FE, Toews GB, White ES. 2008. An essential role for fibronectin extra type III domain A in pulmonary fibrosis. Am J Respir Crit Care Med 177:638-645.