Rett syndrome is a rare genetic neurological disorder that occurs primarily in girls, eventually robbing them of the ability to speak, walk or use their hands, among other devastating effects.

Representatives from UMass Chan presented on several topics and career paths, including RNA Therapeutics Institute, ScienceLive, community and government relations, diversity and inclusion and media and arts in sciences.

An antisense therapy developed by Joel D. Richter, PhD, Sneha Shah, PhD, and Jonathan K. Watts, PhD, at UMass Chan Medical School and Elizabeth Berry-Kravis, MD, PhD, at RUSH University Medical Center, restores production of the protein FMRP in cell samples taken from patients with fragile X syndrome. 

A multidisciplinary team of UMass Chan scientists has developed a technology for delivering siRNA molecules locally to lung tissue, providing the first evidence of a platform capable of delivering chemically modified siRNAs to the lungs nasally and achieving robust genetic silencing.

PhD candidate David Keener has received a National Institutes of Health grant to fund his research project on Rett syndrome, a genetic neurodevelopmental disease generally diagnosed in girls ages 6 to 18 months.

Robert H. Brown Jr., DPhil, MD, Jonathan Watts, PhD, and colleagues showed the ability to suppress mutant forms of the ALS gene known as C9ORF72 in a single-patient pilot study. The paper reporting the results is part of the 2022 STAT News STAT Madness competition.

Robert H. Brown Jr., DPhil, MD, Jonathan Watts, PhD, and colleagues have shown the ability to suppress mutant forms of an ALS gene in a single-patient pilot study.

Six UMass Medical School scientists are among the members of the National Institutes of Health’s Somatic Cell Genome Editing Consortium to publish a paper in Nature outlining the program’s goals.

The new vaccines by Pfizer/BioNTech and Moderna use messenger RNA to stimulate the immune system to protect against COVID-19. These vaccines are the first of their kind and researchers at UMass Medical School are among the leading RNA biologists in the world.