Chapter title |
In Vitro Assays to Assess Exon Skipping in Duchenne Muscular Dystrophy.
|
---|---|
Chapter number | 20 |
Book title |
Cell-Penetrating Peptides
|
Published in |
Methods in molecular biology, January 2015
|
DOI | 10.1007/978-1-4939-2806-4_20 |
Pubmed ID | |
Book ISBNs |
978-1-4939-2805-7, 978-1-4939-2806-4
|
Authors |
Prisca Boisguerin, Liz O’Donovan, Michael J. Gait, Bernard Lebleu |
Editors |
Ülo Langel |
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. |
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Unknown | 14 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
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Researcher | 3 | 21% |
Student > Bachelor | 1 | 7% |
Lecturer > Senior Lecturer | 1 | 7% |
Lecturer | 1 | 7% |
Other | 1 | 7% |
Unknown | 4 | 29% |
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Chemistry | 3 | 21% |
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Unknown | 4 | 29% |