Abstract
Duchenne muscular dystrophy (DMD) is a lethal muscle disorder characterized by mutations in the DMD gene. These mutations primarily disrupt the reading frame, leading to an absence of functional dystrophin protein. Exon-skipping through the use of antisense oligonucleotides has served as a promising therapeutic approach for DMD, and clinical trials in DMD patients are currently underway. Recently, stable and less-toxic antisense oligonucleotides have been developed with a higher efficacy in mouse and dog models of DMD. Despite these advancements, this therapeutic approach is limited by relatively poor systemic delivery of antisense oligonucleotides to muscle, as well as toxicity effects. This review highlights the challenges for antisense oligonucleotide-based therapeutics for DMD, in particular with methods using exon 51-skipping.
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
