Study could lead to advancements in rare disease treatment
A new study has solved the old enigma in mitochondrial DNA disorders and advances the prospect of enhanced treatments for patients.
An international team, some of which are supported by the NIHR Newcastle Biomedical Research Centre (BRC), have published an exciting report in Nature Communications. Their study findings show the identification of small molecules that purge cells of mutant mitochondrial DNA molecules, thereby restoring mitochondrial function.
Exceptionally, this is akin to a ‘gene therapy’ with small molecules. The findings are of considerable importance as they represent a possible treatment for the future.
Professor Robert Taylor, from the Wellcome Centre for Mitochondrial Research, Newcastle University is one of the experts involved in this research, supported by Newcastle BRC. He said:
In identifying the cellular metabolic constraints that can influence mitochondrial DNA replication, this ground-breaking study not only resolves a long-standing conundrum in the molecular pathology of mitochondrial disease, but offers an exciting opportunity for rapid translation of this discovery into real patient benefit through repurposing a compound that has already been tested in human subjects.
Information adapted, with thanks from the Newcastle University Press Office. Read the full story on Newcastle University website