RNA Modification Offers Hope for Treating Neurological Diseases

The research team led by Yongchao C. Ma, Ph.D., at the Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago, uncovered a fundamental biological mechanism that holds promise for the development of novel treatments for neurological disorders like spinal muscular atrophy (SMA) and autism, as well as various types of cancer. Their findings were published in the journal Human Molecular Genetics.

The team led by Dr. Ma discovered that mitochondrial function is regulated by RNA methylation, a chemical modification of RNA. The primary function of mitochondria is to produce energy within cells. Dr. Ma clarifies that mitochondria are capable of much more.

Moreover, it serves as a hub for signals that control several major biological functions in the cell. Previous research conducted by Dr. Ma's lab connected mitochondrial dysfunction to the onset of autism and SMA, while other research has connected it to the pathogenesis of cancer.

Our finding establishes a critical link between RNA methylation, mitochondria, and diseases that relate to mitochondrial dysfunction, which means that now we have potential for new treatments for many different disorders.”

Yongchao C. Ma, Study Senior Author and Associate Professor, Department of Pediatrics, Neurology, and Neuroscience, Feinberg School of Medicine, Northwestern University

Dr. Ma holds the Children's Research Fund Endowed Professorship in Neurobiology at Lurie Children’s.

According to research conducted by Dr. Ma’s group, RNA methylation controls the synthesis of important enzymes that are found inside mitochondria, hence regulating mitochondrial function. They showed that loss of RNA methylation dramatically altered mitochondrial function in stem cells and neurons using models of neural stem cells and mice.

We are very excited about this discovery and the promise of innovative treatments, which could involve developing modifiers of RNA methylation to rectify the mitochondrial defect. We are also honored to contribute significantly to the study of RNA methylation. There is exponential growth in this field, and we hope that our ongoing research on RNA methylation in the nervous system will bring new insights on brain development and neurological disorders.”

Yongchao C. Ma, Study Senior Author and Associate Professor, Department of Pediatrics, Neurology, and Neuroscience, Feinberg School of Medicine, Northwestern University