Electroporation Enhanced Effect of Dystrophin Splice Switching PNA Oligomers in Normal and Dystrophic Muscle

• Autori principali: Camilla Brolin (Autore), Takehiko Shiraishi (Autore), Pernille Hojman (Autore), Thomas O Krag (Autore), Peter E Nielsen (Autore), Julie Gehl (Autore)
• Natura: Libro
• Pubblicazione: Elsevier, 2015-01-01T00:00:00Z.
• Soggetti: article
• Accesso online: Connect to this object online.

Peptide nucleic acid (PNA) is a synthetic DNA mimic that has shown potential for discovery of novel splice switching antisense drugs. However, in vivo cellular delivery has been a limiting factor for development, and only few successful studies have been reported. As a possible modality for improvem...