Search Close Search
Search Close Search

Supporting the boldest research ideas in the interdisciplinary labs under the direction of Robert H. Brown Jr., DPhil, MD, one of the world's foremost authorities on ALS.

2025 Boston Marathon presented by Bank of America

This year's event will take place on Monday, April 21, 2025, and the UMass ALS Cellucci Fund is looking for runners!

The UMass ALS Cellucci Fund is now accepting applications for charity runners who commit to raising at least $7,500 each for ALS research at UMass Chan Medical School. All applications must be completed and returned by Wednesday, Nov. 13, 2024, for consideration.



Submit your application

 brown-watts-crop.jpg

UMass Chan clinical trial shows antisense oligonucleotide safely suppresses mutant ALS gene in pilot human study

Using a short, synthetic chain of chemically modified nucleotides engineered in the RNA Therapeutics Institute at UMass Chan Medical School, Robert H. Brown Jr., DPhil, MD, Jonathan Watts, PhD, and colleagues have shown the ability to suppress mutant forms of the ALS (amyotrophic lateral sclerosis) gene known as C9ORF72 in a single-patient pilot study. C9ORF72 is the most common cause of familial ALS and familial frontotemporal dementia (FTD). The results, published in Nature Medicine, have the potential to catalyze research into treatments for ALS, FTD and other neurodegenerative diseases.

Read more

Page Menu