Evaluation of cell-penetrating peptide delivery of antisense oligonucleotides for therapeutic efficacy in spinal muscular atrophy
Antisense oligonucleotides (ASOs) are a widely used form of gene therapy, which is translatable to multiple disorders. A major obstacle for ASO efficacy is its bioavailability for in vivo and in vitro studies. To overcome this challenge we use cell-penetrating peptides (CPPs) for systemic delivery o...
| Главные авторы: | , , , |
|---|---|
| Формат: | Journal article |
| Язык: | English |
| Опубликовано: |
Springer
2019
|