Single-patient pilot study shows the ability of antisense oligonucleotide to suppress mutant ALS gene

Using a synthetic short chain of chemically modified nucleotides engineered at the Institute of RNA Therapeutics at UMass Chan School of Medicine, Robert H. The ALS gene known as C9ORF72 in a pilot study of a single patient. C9ORF72 is the most common cause of familial amyotrophic lateral sclerosis (ALS) and familial frontotemporal dementia (FTD). … Read more