Researcher of the Gene Therapy and Regulation of Gene Expression Program.
Preferential dedication to gene therapy liver diseases, animal models of liver diseases.
Degree in Biology from the University of the Basque Country. She received her PhD from the Universidad Autónoma de Madrid after completing her doctoral thesis at the Centro de Investigaciones Biológicas del Consejo Superior de Investigaciones Científicas, Madrid, under the direction of research professor Vicente Larraga.
After his PhD, he worked in the group of Dr Ruth Nussenzweig and Dr Moriya Tsuji at New York University, pioneering researchers in the search for new vaccines and treatments against malaria. After three and a half years of stay, she returned to Spain to join the Department of Gene Therapy and Hepatology, of Professor Jesús Prieto, with a Ramón y Cajal contract to direct the Gene Therapy of Viral Hepatitis laboratory, at CIMA of the Universidad de Navarra.
From 2014 to 2019, she served as director of the gene therapy and gene expression regulation program and in November 2018, she moved to the position of deputy director of CIMA. She is currently the coordinator of the area of advanced therapies and diagnostic innovation at the Institute for Health Research of Navarra IdisNA. She is co-founder and scientific director of vivet Therapeutics gene therapy company created in 2016.
She is currently the elected president of the Spanish Society of Gene and Cell Therapy and is on the board of the European Society of Gene and Cell Therapy.
Co-inventor of 20 patents, some of them licensed to biotechnology and pharmaceutical companies, which have led or will lead to clinical trials.
It has regional, national and international, public and private funding.
The coordination of a European Horizon 2020 project thanks to which the first clinical trial of gene therapy for a hereditary metabolic liver disease was carried out.
AREAS OF INTEREST
- Gene therapy for rare hereditary diseases.
- Development of gene therapy vectors and strategies to modulate the immune response against them.
- Study of the mechanisms involved in the development of hereditary and acquired liver diseases.
- Development of animal models of viral hepatitis, study of the host-host relationship.
As an educator
Supervision of more than 20 bachelor's and master's degree theses.
Direction of 16 doctoral theses.
Professor of the following Masters:
- Master in Advanced Therapies at the University Francisco de Victoria.
- Master in Hereditary Metabolic Diseases at the University of Santiago de Compostela.
- Master in Clinical Genetics at the University of Zaragoza.
As a researcher
Shee has participated in the publication of more than 110 scientific articles in international journals.
She has presented more than 100 communications in national and international congresses related to her specialty.
Co-founder of the company Vivet Therapeutics and Scientific Director.
- NATO Postdoctoral Fellowship.
- Basque Government Postdoctoral Fellowship.
- Ramón y Cajal Contract
- Obtained the I3 mention for research excellence awarded by the Ministry of Research and Science.
- Coordination of the European Horizon 2020 project AIPGENE, thanks to which the first clinical trial of gene therapy for a hereditary metabolic liver disease was carried out.
- Foundation of the French-based gene therapy company Vivet therapeutics, whose first gene therapy product for the treatment of Wilson's disease was developed at CIMA and whose safety and efficacy will be tested in patients in 2021.
- Launch of gene therapy research for Dravet disease and development of a gene therapy product for Parkinson's disease licensed to Handl Therapeutics.
- Spanish Society of Gene and Cell Therapy (SETGyC), European Society of Gene and Cell Therapy (ESGCT), American Society of Gene and Cell therapy (ASGCT), European Association for the Study of the Liver (EASL).
- President of the Spanish Society of Gene Therapy 2019-2021. Member of the board of the European Society of Gene Therapy. 2019-2021
Latest scientific publications
- AAV-HDV: An Attractive Platform for the In Vivo Study of HDV Biology and the Mechanism of Disease Pathogenesis Apr 28, 2021 | Magazine: Viruses
- Use of an AAV serotype Anc80 to provide durable cure of phenylketonuria in a mouse model Apr 25, 2021 | Magazine: Journal of Inherited Metabolic Disease