Publications by authors named "Gat Pozner"
Article Synopsis
- Patients with single large-scale mitochondrial DNA (mtDNA) deletion syndromes (SLSMDs) often suffer from serious multisystemic diseases like Pearson syndrome in childhood or Kearns-Sayre syndrome later on, and there's currently no effective treatment available.
- A new approach called mitochondrial augmentation therapy (MAT) was tested on six patients with SLSMDs, where their own hematopoietic cells were infused with healthy maternal mitochondria.
- The treatment showed safety, decreased harmful mtDNA levels in four patients, and increased mtDNA content in all six, alongside some improvements in aerobic function and quality of life measured by caregivers, suggesting a need for further clinical trials.
Mitochondria are cellular organelles critical for numerous cellular processes and harboring their own circular mitochondrial DNA (mtDNA). Most mtDNA associated disorders (either deletions, mutations, or depletion) lead to multisystemic disease, often severe at a young age, with no disease-modifying therapies. Mitochondria have a capacity to enter eukaryotic cells and to be transported between cells.
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