Citation:
Methods Mol Biol. 2015;1324:317-29. doi: 10.1007/978-1-4939-2806-4_20
Abstract:
Cell-penetrating peptide (CPP)-mediated delivery of phosphorodiamidate morpholino oligomers (PMO) results in efficient exon skipping and has shown great promise as a potential therapy for Duchenne muscular dystrophy (DMD). However, large differences in efficiency have been observed between CPPs and in delivery to different tissues. Cellular trafficking has appeared to be an important determinant of activity. This chapter provides details of experimental procedures to monitor exon skipping efficiency and cellular trafficking of Pip6a-PMO, a recently developed and particularly efficient conjugate, in skeletal H2k cells and in primary cardiomyocytes from mdx mice. Similar procedures may be used in principle to evaluate any free or vector-associated oligonucleotide for exon skipping.
Epub:
Not Epub
Link to Publication:
http://link.springer.com/protocol/10.1007%2F978-1-4939-2806-4_20
Organism or Cell Type:
cell culture: skeletal H2k cells and primary cardiomyocytes from mdx mice
Delivery Method:
peptide-linked