Mitochondria-Enriched Stem Cells for Primary Mitochondrial Disease
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Solution Overview
Problem
There is a need for novel methods to increase mitochondrial function in cells and organs affected by various primary mitochondrial diseases and disorders, as existing treatments are cumbersome and not always effective.
Innovation Solution
The use of mammalian stem cells, particularly human stem cells, enriched with exogenous functional mitochondria, which are administered to patients to improve mitochondrial function and treat primary mitochondrial diseases, eliminating the need for lengthy donor matching and lifelong immunosuppression.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If conventional mitochondrial disease treatments are used, then disease management is attempted, but treatment is cumbersome and not always effective
Solution Approach 1:
The patent extracts healthy functional mitochondria from donor cells and introduces them into patient cells to replace defective mitochondria. This extraction and replacement approach directly addresses the unreliable conventional treatments by providing a targeted, effective intervention that specifically replaces the dysfunctional component (mitochondria) rather than attempting general disease management.
Solution Approach 2:
The patent uses stem cells as an intermediary vehicle to deliver healthy mitochondria to target tissues. The stem cells serve as a mediator that protects and transports the therapeutic mitochondria, enabling effective delivery while simplifying the overall treatment approach compared to direct mitochondrial transplantation methods.
2Reliability
If allogeneic stem cell transplantation is performed, then mitochondrial function can be improved, but donor matching and immunosuppression are required
Solution Approach 1:
The patent extracts only the functional mitochondria from donor stem cells and transfers them to patient cells, leaving behind the cell nucleus and other components that would trigger immune rejection. This extraction approach enables mitochondrial replacement without requiring full donor matching or lifelong immunosuppression, significantly reducing the time loss associated with donor screening and matching.
Solution Approach 2:
The patent applies local quality by transferring only the specific functional component (mitochondria) needed for therapy while leaving other cellular components in the patient's own cells. This localized approach to mitochondrial replacement avoids the immune compatibility issues that would require extensive donor matching procedures.
3Reliability
If high levels of mitochondrial enrichment are achieved, then therapeutic effect is maximized, but processing complexity increases
Solution Approach 1:
The patent employs partial action by achieving moderate mitochondrial enrichment levels (not complete replacement) that are sufficient to provide therapeutic benefit. This approach maximizes therapeutic effect while avoiding the excessive processing complexity that would be required to achieve 100% mitochondrial replacement, demonstrating that partial enrichment is adequate for clinical improvement.
Data Source
AI summary
The present invention provides human stem cells enriched with functional mitochondria, wherein the healthy functional exogenous mitochondria constitute at least 3% and less than 33% of the total mitochondria in the mitochondrially-enriched human stem cells. Further disclosed are methods for the production of such cells, and uses thereof for the treatment of primary mitochondrial diseases.