Link between MHC Fiber Type and Restoration of Dystrophin Expression and Key Components of the DAPC by Tricyclo-DNA-Mediated Exon Skipping

• मुख्य लेखकों: Saleh Omairi (लेखक), Kwan-Leong Hau (लेखक), Henry Collin-Hooper (लेखक), Federica Montanaro (लेखक), Aurelie Goyenvalle (लेखक), Luis Garcia (लेखक), Ketan Patel (लेखक)
• स्वरूप: पुस्तक
• प्रकाशित: Elsevier, 2017-12-01T00:00:00Z.
• विषय: article
• ऑनलाइन पहुंच: Connect to this object online.

Exon skipping mediated by tricyclo-DNA (tc-DNA) antisense oligonucleotides has been shown to induce significant levels of dystrophin restoration in mdx, a mouse model of Duchenne muscular dystrophy. This translates into significant improvement in key disease indicators in muscle, cardio-respiratory...