A gene therapy to correct a mutation that causes maple syrup urine disease prevented newborn death and normalized growth in a calf as well as in mice.

Overall, there’s more work to do. But new mitochondrial base editors “should help improve the precision of mitochondrial gene therapy,” the team wrote.

More information: Kai Chen et al, mLumiOpto Is a Mitochondrial-Targeted Gene Therapy for Treating Cancer, Cancer Research (2024). DOI: 10.1158/0008-5472.CAN-24-0984 ...

Maintaining mitochondrial DNA (mtDNA) integrity is crucial for cardiomyocyte function, and its disruption plays a significant role in ischemia/reperfusion (I/R) injury.

Discover how hyperbaric oxygen therapy influences telomeres, stem cells, and mitochondria. New research reveals promising ...

Liu and Zhou are working with clinicians Herbert Chen, M.D., chair, Department of Surgery and Eddy Yang, M.D., Ph.D., professor, Department of Radiation Oncology, to fully investigate the therapeutic ...

The therapy was designed with the aim of boosting mitochondrial activity (mitochondria are "cellular energy generators" ...

“The authors were able to optimize pronuclear transfer to a point where mitochondrial carryover was not a common problem,” he told The Scientist. “But this is exactly the kind of data we need more of ...

is in Phase III trials in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based ...