With a $90,000 grant from FRAXA Research Foundation, Dr. Mollie Meffert and Dr. Christina Timmerman studied groups of small RNAs, known as microRNAs, which are greatly decreased in brain tissue of fragile X mice vs. normal controls.
The search for blood-based biomarkers in fragile X has been a topic of intense interest in recent years, and is seen by many in the field as essential to the development of new treatments. While most of the effort has focused on examination of proteins which are significantly increased or decreased in fragile X, this project takes a very different approach. Dr. Mollie Meffert, Dr. Christina Timmerman, and colleagues are looking at groups of small RNAs, known as microRNAs, which are greatly decreased in brain tissue of fragile X mice vs. normal controls. These microRNAs do not code for any protein (unlike messenger RNA) but are important regulatory elements. MicroRNAs are also easily measurable in blood, and generally highly stable relative to proteins or longer RNAs, making them attractive as candidate biomarkers.
Christina Timmerman, PhD