Research by Victor Ambros, PhD, provides new insights into a recently identified class of neurodevelopmental disorders called Argonaute syndromes. Dr. Ambros and colleagues believe their Proceedings of the National Academy of Sciences paper will help inform clinical and translational researchers in their pursuit of a treatment for this class of neurodevelopmental and autism-spectrum disorders.
“These findings will help the clinical scientists studying Argonaute syndromes in human cells to test for similar biological mechanisms,” said Ambros, the Silverman Chair in Natural Sciences and professor of molecular medicine. “Much of basic biology is shared between organisms. The power of the C. elegans model is that we can learn something new about basic biology that can then be applied directly to humans and human disease. Our work uncovers potential new aspects of Argonaute function in humans that then can help guide the search for treatments for disease.”
A central figure in ribonucleic acid (RNA) biology research, Ambros was the first person to identify and characterize a known microRNA, a class of noncoding RNAs that plays an important role in modulating or regulating gene expression, specifically the process by which genes are silenced or turned off. Today, the discovery of microRNA is recognized as a pioneering step toward understanding that many RNA molecules play a critical role in the complex regulation of genes.
