In Vitro Studies to Evaluate the Intestinal Permeation of an Ursodeoxycholic Acid-Conjugated Oligonucleotide for Duchenne Muscular Dystrophy Treatment

• Main Authors: Marika Faiella (Author), Giada Botti (Author), Alessandro Dalpiaz (Author), Lorenzo Gnudi (Author), Aurélie Goyenvalle (Author), Barbara Pavan (Author), Daniela Perrone (Author), Matteo Bovolenta (Author), Elena Marchesi (Author)
• Format: Book
• Published: MDPI AG, 2024-08-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Delivery represents a major hurdle to the clinical advancement of oligonucleotide therapeutics for the treatment of disorders such as Duchenne muscular dystrophy (DMD). In this preliminary study, we explored the ability of 2'-<i>O</i>-methyl-phosphorothioate antisense oligonucleotid...