A Short Antisense Oligonucleotide Ameliorates Symptoms of Severe Mouse Models of Spinal Muscular Atrophy

• Main Authors: Jeffrey M Keil (Author), Joonbae Seo (Author), Matthew D Howell (Author), Walter H Hsu (Author), Ravindra N Singh (Author), Christine J DiDonato (Author)
• Format: Book
• Published: Elsevier, 2014-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Recent reports underscore the unparalleled potential of antisense-oligonucleotide (ASO)-based approaches to ameliorate various pathological conditions. However, in vivo studies validating the effectiveness of a short ASO (<10-mer) in the context of a human disease have not been performed. One dis...