Leber's Hereditary Optic Neuropathy (LHON) is a genetic disorder that leads to vision loss and blindness, primarily due to mutations in mitochondrial DNA (mtDNA) that impair the function of the respiratory chain.

In a study published in Science China Life Sciences, a team of researchers led by Prof. LIU Xingguo from the Guangzhou Institutes of Biomedicine and Health of the Chinese Academy of Sciences and Prof. ZHANG Qingjiong from Zhongshan Ophthalmic Center, discovered that mesenchymal stem cell (MSC)-mediated mitochondrial transfer can effectively restore mtDNA and improve mitochondrial function in neural progenitor cells derived from patients with LHON.

The researchers reprogrammed urine cells from LHON patients into induced pluripotent stem cells (iPSCs), and subsequently differentiated them into neural progenitor cells. By co-culturing these neural progenitor cells with MSCs, they observed significant improvements in mitochondrial function and an increase in the proportion of normal mtDNA.

Patch-clamp recordings demonstrated that LHON neurons, which typically exhibit impaired sodium and potassium currents, showed restored electrophysiological function after co-culture with MSCs. Specifically, both inward sodium and outward potassium currents were significantly enhanced in LHON neurons following the mitochondrial transfer from MSCs.

The results suggested 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.