Antisense Transcription

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

A UMass Chan clinical trial demonstrates the safety and efficacy of an antisense oligonucleotide in suppressing mutant C9ORF72, a common cause of familial ALS. The treatment led to reduced levels of neurotoxins and stable or improved ALS functional scores.