Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle
oleh: James S. Novak, Marshall W. Hogarth, Jessica F. Boehler, Marie Nearing, Maria C. Vila, Raul Heredia, Alyson A. Fiorillo, Aiping Zhang, Yetrib Hathout, Eric P. Hoffman, Jyoti K. Jaiswal, Kanneboyina Nagaraju, Sebahattin Cirak, Terence A. Partridge
| Format: | Article |
|---|---|
| Diterbitkan: | Nature Portfolio 2017-10-01 |
Deskripsi
Exon skipping is a strategy for the treatment of Duchenne muscular dystrophy, but has variable efficacy. Here, the authors show that dystrophin restoration occurs preferentially in areas of myofiber regeneration, where antisense oligonucleotides are stored in macrophages and delivered to myoblasts and newly formed myotubes