User talk:Tindle Lisa/My sandbox Support page v2

                

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User talk:Tindle Lisa/My sandbox Support page v2

"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

WP2 - Name of person and/or instution responsible for this workpackage

• Bullet points of WP objectives

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.