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Our Science

Open Questions

Pearson and KSS are non-inherited mitochondrial disease. Therefore, we can use the mother as a donor of mitochondria. Other diseases are inherited from mothers, can we use allogeneic mitochondria from donors?

We intend to use placenta as a source of allogeneic mitochondria to create a bank of ready-to-use frozen mitochondria. We need to show that indeed allogeneic mitochondria can be used, first prove in-vitro with CD34+ cells as well as in-vivo in NSGS mice.

What is the Mode of Action of our therapy? Do CD34+ cells home to damaged organs and transfer mitochondria?

Biodistribution study of normal mitochondria in a mutated mouse model; Use new imaging modalities and animal models; analyzing changes in gene expression and cytokine secretion after MAT

Will we need to re-treat the patients? How often? How will we know?

Currently we have only tried single treatment. We will continue to follow-up on all patients, mainly looking on blood heteroplasmy and well-being.

What happens with the damaged mitochondria? Are they cleared away?

Our direct process does not eliminate the damaged mitochondria, only enrich the cells with healthy ones. The hypothesis is that the quality control of the cell maintains the normal mitochondria and eliminate damaged mitochondria through mitophagy. We would like to explore mitophagy after MAT.