Dr Baruteau graduated as a Medical Doctor and Specialist in Paediatrics from Toulouse University in 2004. He specialised in Paediatric Inherited Metabolic Medicine in 3 different tertiary metabolic centres in Paris and Toulouse, France then in London. He was appointed as Consultant in Paediatric Metabolic Medicine at Great Ormond Street Hospital for Children in London in 2017.
Dr Baruteau is a clinical academic with a strong commitment to basic and clinical research. He is a Group Leader at University College London Great Ormond Street Institute of Child Health. He has been awarded prestigious fellowships to pursue his clinical and academic career: Action Medical Research Training Fellowship, NIHR GOSH Biomedical Research Centre Catalyst Fellowship, MRC Clinician Scientist Fellowship. He was promoted as Principal Research Fellow (Equivalent of Associate Professor) in 2022. His research focuses on studying the pathophysiology and developing novel therapies for inherited metabolic diseases. He has developed gene therapy using both viral and non-viral vectors and is the UK chief investigator in first-in-human gene therapy clinical trial. He has expertise in translating academic and industry-led programmes of gene therapies. He is the member of experts’ committees from the European Society of Gene and Cell Therapy, the American Society of Gene and Cell Therapy and the European Society of Human Genetics. He has served as expert for the French medicine agency “Agence nationale du médicament et des produits de santé” for 10 years. He has authored >70 scientific articles.
Dr Baruteau is an invited member of the scientific board of the UK-based Children’s Liver Disease Foundation.
- English, French, Portuguese (Basic)
Disorders of intermediary metabolism
Urea cycle defects
Lysosomal storage diseases
Paediatric training in Toulouse & Nantes, France
Sub-specialty training in Inherited Metabolic Diseases in Paris & Toulouse, France and London (UK)
Master of Science (MSc) from Toulouse University with laboratory training in Prof Sokal’s laboratory in Brussels, Belgium
PhD in Gene Therapy from University College London. Supervision by Prof Waddington, Gissen and Mills.
Fellow of the Royal College of Paediatrics and Child Health
Invited member of organising committees from the American Society of Gene and Cell Therapy, European Society of Gene and Cell Therapy, European Society of Human Genetics.
Gene therapy with viral (adeno-associated viral (AAV), lentiviral) and non-viral (exosomes, lipid nanoparticles encapsulating mRNA) vectors
Liver inherited metabolic diseases, particularly urea cycle defects
News & Publications
1. Gurung S*, Timmermand OV*, Percoheau DP*, Gil-Martinez AL, Minnion M, Touramanidou L, Fand S, Messina M, Khalil Y, Barber AR, Edwards RS, Finn PF, Siddiqui S, Rice L, Martini PGV, Mills PB, Waddington SN, Gissen P, Eaton S, Ryten M, Feelisch M, Frassetto A, Witney TH**, Baruteau J**. mRNA therapy restores ureagenesis and corrects glutathione metabolism in argininosuccinic aciduria. Science Translational Medicine. In Press. BioRxiv. https://www.biorxiv.org/content/10.1101/2022.10.19.512931v1
2. Elkhateeb N, Olivieri G, Siri B, Stepien KM, Sharma R, Morris A, Crowther L, Grunewald S, Chakrapani A, Lachmann R, Murphy E, Santra S, Uudelepp ML, Yeo M, Mills PO, Ridout D, Gissen P, Dionisi-Vici C, Baruteau J. Natural history of epilepsy in argininosuccinic aciduria provides new insights in pathophysiology: an international retrospective study. Epilepsia. 2023 In Press.
3. Duff C, Alexander IE, Baruteau J. Gene therapy for Urea Cycle Defects: an update from historical perspectives to future prospects. J Inherit Metab Dis. 2023 In Press.
4. Duff C, Baruteau J. Modelling urea cycle disorders using iPSCs. NPG Regen Med. 2022; 7:56.
5. Baruteau J, Cunningham SC, Seker B, Perocheau DP, Eaglestone S, Thrasher AJ, Waddington SN, Lisowski L, Alexander IE, Gissen P. Safety, efficacy and biodistribution of an engineered hepatotropic Adeno-Associated Virus mediated gene therapy for Ornithine Transcarbamylase Deficiency in Cynomolgus monkeys. Mol Ther Meth Clin Dev. 2021;23:135-146
6. Soria LR, Gurung S, De Sabbata G, Perocheau DP, De Angelis A, Bruno G, Polishchuk E, Paris D, Cuomo P, Motta A, Orford M, Khalil Y, Eaton S, Mills PB, Waddington SN, Settembre C, Muro A, Baruteau J, Brunetti-Pierri N. Beclin-1-mediated activation of autophagy improves proximal and distal urea cycle disorders. EMBO Mol Med 2021:13(2):e13158.
7. Baruteau J, Diez-Fernandez C, Lerner S, Ranucci G, Gissen P, Dionisi-Vici C, Nagamani S, Erez A, Häberle J. Argininosuccinic aciduria: recent pathophysiological insights and therapeutic prospects. J Inherit Metab Dis. 2019;42(6):1147-1161.
8. Baruteau J, Waddington, SN. Fetal gene therapy for neurodegenerative lysosomal storage diseases. J Inherit Metab Dis. 2019;42(3):391-393.
9. Baruteau J, Perocheau DP, Hanley J, Lorvellec M., Rocha-Ferreira E, Karda R, Ng J, Suff N, Diaz JA, Rahim AA, Hughes MP, Banushi B, Prunty H, Hristova M, Ridout DA, Virasami A, Heales S, Howe SJ, Buckley SMK, Mills PB, Gissen P, Waddington SN. Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer. Nat Commun. 2018;9:3505.
10. Baruteau J, Jameson E, Morris AA, Chakrapani A, Santra S, Vijay S, Grunewald S, Murphy E, Cleary M, Mundy H, Abulhoul L, Broomfield A, Lachmann R, Rahman Y, Ridout DA, Waddington SN, Mills PB, Gissen P, Davison J. Expanding the phenotype in argininosuccinic aciduria: need for new therapies. J Inherit Metab Dis. 2017 May;40(3):357-368.
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