Academic Journals Database
Disseminating quality controlled scientific knowledge

Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy

ADD TO MY LIST
 
Author(s): Goyenvalle Aurélie | Davies Kay E

Journal: Skeletal Muscle
ISSN 2044-5040

Volume: 1;
Issue: 1;
Start page: 8;
Date: 2011;
Original page

ABSTRACT
Abstract Antisense oligonucleotides are short nucleic acids designed to bind to specific messenger RNAs in order to modulate splicing patterns or inhibit protein translation. As such, they represent promising therapeutic tools for many disorders and have been actively developed for more than 20 years as a form of molecular medicine. Although significant progress has been made in developing these agents as drugs, they are yet not recognized as effective therapeutics and several hurdles remain to be overcome. Within the last few years, however, the prospect of successful oligonucleotides-based therapies has moved a step closer, in particular for Duchenne muscular dystrophy. Clinical trials have recently been conducted for this myopathy, where exon skipping is being used to achieve therapeutic outcomes. In this review, the recent developments and clinical trials using antisense oligonucleotides for Duchenne muscular dystrophy are discussed, with emphasis on the challenges ahead for this type of therapy, especially with regards to delivery and regulatory issues.
Why do you need a reservation system?      Affiliate Program