Watts Lab News

UMass Chan scientists have received funding to explore gene editing technologies for treating frontotemporal dementia.

UMass Chan licenses potential RNA-based treatment for fragile X syndrome to QurAlis Corporation.

UMass Chan supports translational research from faculty with internal funding mechanism.

UMass Chan Medical School and QurAlis Corporation, a clinical-stage biotechnology company investigating treatments for amyotrophic lateral sclerosis, frontotemporal dementia and other neurodegenerative and neurological diseases, have formed a collaboration to investigate an antisense oligonucleotide technology for the potential treatment of fragile X syndrome.  

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.