Antisense Therapy Safely Dampens Mutant C9orf72 in ALS Patient
An experimental antisense oligonucleotide that works to suppress the mutant C9orf72 gene — a cause of amyotrophic lateral sclerosis (ALS) — safely lowered the production of damaging proteins and other molecules in a patient in a pilot trial. “While other teams have documented that this gene can be suppressed in cells…