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 an ALS gene known as C9ORF72 in a single-patient pilot study.
Amyotrophic lateral sclerosis, known as ALS, is a neurodegenerative disorder which affects motor neurons. These are the neuronal cells responsible for muscle innervation, whose degenerations leads to progressive paralysis culminating with motor and respiratory failure.