Weeden et al. use a method called FORCE to deliver oligonucleotides to muscle and evaluate the platform’s utility in models of myotonic dystrophy. An antisense oligonucleotide targeting DMPK is guided to muscle tissue with an antibody fragment and beneficial results are seen in mouse models of disease and cynomolgus monkey.
- Timothy Weeden
- Tyler Picariello
- Stefano Zanotti
