A new report by Dr. Alex MacKenzie, MD, PhD, of the Children’s Hospital of Eastern Ontario and the University of Ottawa, has been published in the New England Journal of Medicine (N Engl J Med 2012; 366:761-763; 02/23/2012).
The report, entitled “Sense in Antisense Therapy for Spinal Muscular Atrophy”, discusses recent understandings on the use of antisense oligonucleotide (or, ‘ASO’) therapy for orphan genetic disease, stating: “…ASO now joins and may overtake gene therapy as the leading experimental approach to the treatment of spinal muscular atrophy.”
You can read the entire publication (as a pdf) right here: Sense in Antisense Therapy for SMA.
Online subscribers can also find it at http://www.nejm.org/doi/full/10.1056/NEJMcibr1114629.
Dr. MacKenzie serves as Scientific Advisory Co-Chair for FightSMA.