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===Aims=== | ===Aims=== | ||
The project aims to use iPSCs and reprogramming-drived drug discovery for finding therapies against MILS. A personalized medicine approach will be applied and compound screenings on patient-derived neural cells will be carried out. Brain organoids and multi-omics analysis will be used for validations. | |||
1. Establishment of PDE5 inhibitors as reference drugs for MILS | 1. Establishment of PDE5 inhibitors as reference drugs for MILS | ||
2. Large-scale screen of marketed compounds in MILS neural cells | 2. Large-scale screen of marketed compounds in MILS neural cells | ||
3. Validations of hit 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 | 4. Validations of hit compounds in neural cells carrying LS-associated Complex I mutations | ||
5. Computational analysis of MILS neural cells and treatments | |||
===Objectives=== | ===Objectives=== | ||
:::: '''WP1''' - Name of person and/or instution responsible for this workpackage | :::: '''WP1''' - Name of person and/or instution responsible for this workpackage | ||
::::* Bullet points of WP objectives | ::::* Bullet points of WP objectives | ||
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== Support == | == Support == | ||
This project has received funding from the European Joint Programme on Rare Diseases (EJP RD). | |||
Funded projects: https://www.ejprarediseases.org/index.php/funded-projects-2020/ |
Revision as of 11:13, 21 December 2020
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
The project aims to use iPSCs and reprogramming-drived drug discovery for finding therapies against MILS. A personalized medicine approach will be applied and compound screenings on patient-derived neural cells will be carried out. Brain organoids and multi-omics analysis will be used for validations. 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.
CureMILS events
CureMILS publications
CureMILS dissemination
Links
Social Media
Research Networks
References
Additional information
Support
This project has received funding from the European Joint Programme on Rare Diseases (EJP RD).
Funded projects: https://www.ejprarediseases.org/index.php/funded-projects-2020/