Drug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural Cells
Recruiting
Observational Study
Leigh Syndrome (Maternally Inherited, MILS)
Leigh Syndrome (AR, AD, XR)
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Sex
- Any
- Study type
- Observational
- Participants needed
- 80 (estimated)
- Sponsor
- Charite University, Berlin, Germany · Other
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About This Trial
In this project, the investigators are using iPSC lines derived from patients with Leigh syndrome that carry mutations in the mitochondrial (mtDNA) and in the nuclear DNA (nDNA) to reprogram them into neural progenitor cells and into dopaminergic neurons. The researchers are using this experimental system to screen FDA (Food and Drug Administration, USA) and EMA (European Medicines Agency) approve…
Trial Locations
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Eligibility Criteria
Inclusion Criteria: 1. Patient has a disease causing mutation in one of the genes causing Leigh syndrome if mutated, 2. Patient has the characteristic cranial MRI abnormalities of Leigh syndrome
Exclusion Criteria: 1. bleeding disorder that precludes a skin biopsy, 2. retraction of consent
Contacts