Intramuscular Evaluation of Chimeric Locked Nucleic Acid/2'<i>O</i>Methyl-Modified Antisense Oligonucleotides for Targeted Exon 23 Skipping in Mdx Mice

• Main Authors: Michaella Georgiadou (Author), Melina Christou (Author), Kleitos Sokratous (Author), Jesper Wengel (Author), Kyriaki Michailidou (Author), Kyriacos Kyriacou (Author), Andrie Koutsoulidou (Author), Nikolaos P. Mastroyiannopoulos (Author), Leonidas A. Phylactou (Author)
• Format: Book
• Published: MDPI AG, 2021-10-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Duchenne muscular dystrophy (DMD) is a fatal disorder characterised by progressive muscle wasting. It is caused by mutations in the dystrophin gene, which disrupt the open reading frame leading to the loss of functional dystrophin protein in muscle fibres. Antisense oligonucleotide (AON)-mediated sk...