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

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.

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.

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

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 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 ...

This complementation shows a mitochondria-specific mechanism for avoiding expression of deletion-mutant mtDNA, and opens the possibility of a gene therapy in which mitochondria possessing full ...

Mitochondrial gene products interact with those encoded in nuclear genes, and sometimes with the nuclear genome itself. Because the mitochondrial genome mutates faster than the nuclear genome, it ...

Researchers from the Mitochondrial Medicine Program at ... For example, this study found that a recurrent deleted region within the gene MT-ND5 occurred in 96% of SLSMD patients, regardless ...

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 ...