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"A reprogramming-based strategy for drug repositioning in patients with mitochondrial DNA-associated Leigh syndrome (MILS)"
"Project Name": "Funding Scheme"
- EJP RD JTC 2020: "PRE-CLINICAL RESEARCH TO DEVELOP EFFECTIVE THERAPIES FOR RARE DISEASES” - Project EJPRD20-010
- Duration: 36 months
- Start:
- Web:
About CureMILS
- Led by Prof. Dr. Alessandro Prigione of the Heinrich Heine University, the CureMILS project aims to employ a novel approach to enable drug discovery of MILS.
Aims
1. Establishment of PDE5 inhibitors as reference drugs for MILS 2. Large-scale screen of marketed compounds in MILS neural cells
3. Validations of hit compounds in MILS neural cells
4. Validations of hit compounds in neural cells carrying LS-associated Complex I mutations
5. Computational analysis of MILS neural cells and treatments
Objectives
- WP1 - Name of person and/or instution responsible for this workpackage
- Bullet points of WP objectives
- WP1 - Name of person and/or instution responsible for this workpackage
- WP2 - Name of person and/or instution responsible for this workpackage
- Bullet points of WP objectives
- WP2 - Name of person and/or instution responsible for this workpackage
Coordinator
- Alessandro Prigione, MD, PhD - Department of General Pediatrics, Neonatology, and Pediatric Cardiology at Heinrich Heine University, DE - https://www.neurosciences-duesseldorf.de/principal-investigators-and-junior-researchers/alessandro-prigione OR https://www.hhu.de/en/
Network
Participants/Beneficiaries
- Fraunhofer IME, Department ScreeningPort,
- Institute for Molecular Biology, Innsbruck University
- Radboud University Medical Center, Biochemistry
- University of Helsinki, Research Program of Stem Cells and Metabolism
- Institute of Genetics and Animal Biotechnology of the Polish Academy of Sciences, Department of Molecular Biology
- Verona University, Department of Diagnostics and Public Health
- Luxembourg Centre for Systems Biomedicine (LCSB) University of Luxembourg
- International Mito Patients (IMP)
Collaborators
- Charité University, Clinic for Pediatrics and Neurology
- Department of General Pediatrics, Neonatology and Pediatric Cardiology, University Children's Hospital, Heinrich Heine University
- Oroboros Instruments GmbH
- Foundation IRCCS Neurological Institute C. Besta, Unit of Molecular Neurogenetics
- University of Milan, Department of Medical Biotechnology and Translational Medicine
- Max Planck Institute for Molecular Genetics
- Deutsches MITONET e.V., Friedrich-Baur-Institut
Oroboros project involvement
- Oroboros is a collaborator on the project providing infrastructure and expertise required to conduct respirometric measurements for the project.