Efficacy and Safety Profile of Tricyclo-DNA Antisense Oligonucleotides in Duchenne Muscular Dystrophy Mouse Model

• Main Authors: Karima Relizani (Author), Graziella Griffith (Author), Lucía Echevarría (Author), Faouzi Zarrouki (Author), Patricia Facchinetti (Author), Cyrille Vaillend (Author), Christian Leumann (Author), Luis Garcia (Author), Aurélie Goyenvalle (Author)
• Format: Book
• Published: Elsevier, 2017-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Antisense oligonucleotides (AONs) hold promise for therapeutic splice-switching correction in many genetic diseases. However, despite advances in AON chemistry and design, systemic use of AONs is limited due to poor tissue uptake and sufficient therapeutic efficacy is still difficult to achieve. A n...