Locked Nucleic Acid Gapmers and Conjugates Potently Silence ADAM33, an Asthma-Associated Metalloprotease with Nuclear-Localized mRNA

• Main Authors: Hannah M. Pendergraff (Author), Pranathi Meda Krishnamurthy (Author), Alexandre J. Debacker (Author), Michael P. Moazami (Author), Vivek K. Sharma (Author), Liisa Niitsoo (Author), Yong Yu (Author), Yen Nee Tan (Author), Hans Michael Haitchi (Author), Jonathan K. Watts (Author)
• Format: Book
• Published: Elsevier, 2017-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Two mechanisms dominate the clinical pipeline for oligonucleotide-based gene silencing, namely, the antisense approach that recruits RNase H to cleave target RNA and the RNAi approach that recruits the RISC complex to cleave target RNA. Multiple chemical designs can be used to elicit each pathway. W...