Preclinical Studies on Intestinal Administration of Antisense Oligonucleotides as a Model for Oral Delivery for Treatment of Duchenne Muscular Dystrophy

• Main Authors: Maaike van Putten (Author), Courtney Young (Author), Sjoerd van den Berg (Author), Amanda Pronk (Author), Margriet Hulsker (Author), Tatyana G Karnaoukh (Author), Rick Vermue (Author), Ko Willems van Dijk (Author), Sjef de Kimpe (Author), Annemieke Aartsma-Rus (Author)
• Format: Book
• Published: Elsevier, 2014-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Antisense oligonucleotides (AONs) used to reframe dystrophin mRNA transcripts for Duchenne muscular dystrophy (DMD) patients are tested in clinical trials. Here, AONs are administered subcutaneously and intravenously, while the less invasive oral route would be preferred. Oral delivery of encapsulat...