Groundbreaking Discovery Paves Way for New Treatments of Neurological Diseases and Cancer

Researchers at Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago have made a major breakthrough that could lead to new treatments for neurological diseases and cancer. The team, led by Dr. Yongchao C. Ma, found that a chemical change in RNA, called RNA methylation, plays a crucial role in controlling how mitochondria work in cells.

Mitochondria are known for producing energy in cells, but they also act as a control center that regulates many important biological processes. Dr. Ma’s previous research has linked problems with mitochondria to the development of spinal muscular atrophy (SMA) and autism, while other researchers have connected mitochondrial issues to 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,” said Dr. Ma, who holds the Children’s Research Fund Endowed Professorship in Neurobiology at Lurie Children’s and is Associate Professor of Pediatrics, Neurology, and Neuroscience at Northwestern University Feinberg School of Medicine.

The researchers discovered that RNA methylation controls the production of key enzymes that are part of mitochondria. They showed in both stem cells and mice that when RNA methylation was lost, it significantly altered how mitochondria functioned in stem cells and neurons.

This groundbreaking finding opens up exciting possibilities for developing new treatments that target RNA methylation to fix mitochondrial defects. Dr. Ma and his team are enthusiastic about the potential impact of their discovery.

“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,” said Dr. Ma.

The study, published in the journal Human Molecular Genetics, is a significant contribution to the rapidly growing field of RNA methylation research. Dr. Ma and his team at Lurie Children’s are committed to continuing their work on RNA methylation in the nervous system, with the goal of gaining new insights into brain development and neurological disorders.

Lurie Children’s is a top-ranked, nonprofit children’s hospital dedicated to providing exceptional care to every child. The hospital’s research is conducted through Stanley Manne Children’s Research Institute, which focuses on improving child health, transforming pediatric medicine, and ensuring healthier futures through the relentless pursuit of knowledge. Lurie Children’s also serves as the pediatric training ground for Northwestern University Feinberg School of Medicine.

With this remarkable discovery, Dr. Ma and his team have opened the door to a new era of innovative treatments for neurological diseases and cancer, offering hope to countless patients and their families.



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