A Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD in Pediatric Patients With Pearson Syndrome

Inclusion criteria

• Patient diagnosed with Pearson Syndrome, as verified by molecular identification of a defect in the mitochondrial DNA.
• Normal maternal mitochondria as verified by mtDNA sequencing.
• Males and females between 3 years or older and up to 18th birthday.
• Patient is transfusion independent.
• Patient has at least one of the following systematic involvements:

• High baseline lactate levels
• Episodes of metabolic crisis in the last year before pre-screening
• Renal failure (not dependent on dialysis) or evidence of proximal tubulopathy
• Growth retardation or failure to thrive

Exclusion criteria

• Absence of detectable mitochondria mutation or deletion.
• Patient or patient's mother have a positive test for microbiologic
• Patient is unable to undergo leukapheresis.
• Patient suffers from chronic severe infection, malignant disease or any other disease or condition that may risk the patient or interfere with the ability to interpret the study results.
• Patient has been treated previously with any cell or gene therapy.
• Patient has participated in another clinical treatment trial or received other experimental medications outside of a clinical trial within 1 month prior to start of this study.