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?