microRNA (miRNA) Archives

Characterization and Modulation of microRNAs in Fragile X Syndrome

June 7, 2022

MicroRNAs are disrupted in Fragile X; the team will work to understand this and explore ways to correct it with drugs which directly target microRNAs.

MicroRNA Mediated Astroglial GLT1 Dysregulation in Fragile X

September 30, 2017

The team studied how glial cells, especially astrocytes, affect Fragile X. They tested microRNAs to restore GLT1 and reduce excess glutamate linked to brain hyperexcitability.

Trial and No Error: Better Outcomes for Clinical Trials in Fragile X Syndrome

December 14, 2016

Johns Hopkins researcher Christina Timmerman, PhD, searches for a less subjective method to determine if a drug is working in patients with Fragile X syndrome. Many parents of children with Fragile X syndrome were crushed when promising drug trials were unexpectedly stopped a few years ago because subjective behavior-based outcome measures did not justify continuing the trials. The strong feelings linger today. If all goes well with Christina Timmerman’s research, future drug trials may be able to continue with additional metrics for assessment, until there are advanced treatments or even a cure for Fragile X syndrome.

MicroRNAs as Biomarkers in Fragile X Syndrome

September 10, 2016

The team at Johns Hopkins University studied groups of small RNAs, known as microRNAs, which are greatly decreased in brain tissue of Fragile X mice vs. normal controls.