NEALS SOD1 Research is First Antisense Oligonucleotide Trial for a Central Nervous System Disorder

Antisense Oligonucleotides May be Feasible ALS Therapeutic Strategy

BOSTON, Massachusetts, March 29, 2013 — Published online today in Lancet Neurology, an SOD1-related familial ALS trial under the direction of NEALS researchers Timothy Miller, MD, PhD (Washington University School of Medicine) and Merit Cudkowicz (Massachusetts General Hospital) indicates that antisense oligonucleotide delivery to the central nervous system may [...]

2 New Genes Linked to ALS and Related Disorders

Recognition that the mutations adversely impact regulation of RNA could lead to targeted therapy to correct the problem. The mutation’s location in the prion-like domain might also prove significant. Although the mutations in hnRNPA2B1 or hnRNPA1 appear to be rare, hundreds of other RNA-binding proteins have prion-like domains. Taylor said patients with unexplained neurodegenerative diseases may have mutations in these proteins.