Key Takeaways

• Transferring mitochondria from healthy cells to cells from patients with Leber’s hereditary optic neuropathy (LHON), a blinding eye condition caused by defects in mitochondria, improved mitochondrial function in the LHON cells.
• The results provide a promising avenue for future research and clinical applications in treating mitochondrial disorders such as LHON.

Mitochondria, often referred to as the powerhouses of the cell, help turn the energy we take from food into energy the cell can use. While most of the genetic material in a cell is contained the nucleus, mitochondria have their own set of genetic material, called mitochondrial DNA (mtDNA), that is important to their function. Mutations in this DNA can cause disease, such Leber’s hereditary optic neuropathy (LHON), a genetic disorder that leads to vision loss and blindness.

In a recent study published in Science China Life Sciences, scientists reprogrammed urine cells from LHON patients into a type of stem cell called induced pluripotent stem cells (iPSCs) and subsequently differentiated them into neural progenitor cells. They then cultured these cells with another type of cell called mesenchymal stem cells (MSCs) that had healthy mitochondria.

The researchers found that this procedure resulted in significant improvements in mitochondrial function in the LHON cells and an increase in the proportion of normal mtDNA. The results suggest that MSCs can transfer functional mitochondria to neural progenitor cells, thus restoring their function and potentially offering a new therapeutic strategy for mitochondrial diseases. This study provides a promising avenue for future research and clinical applications in treating mitochondrial disorders such as LHON.

Edited by Miriam Kaplan, PhD

Source:

Science China Press, Medical Xpress, August 29, 2024; see source article