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

• Main Authors: Camilla Brolin (Author), Takehiko Shiraishi (Author), Pernille Hojman (Author), Thomas O Krag (Author), Peter E Nielsen (Author), Julie Gehl (Author)
• 格式: 图书
• 出版: Elsevier, 2015-01-01T00:00:00Z.
• 主题: article
• 在线阅读: 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...