Antisense Oligonucleotides (ASOs) Products for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder characterized by progressive muscle degeneration. The disease is generally caused by loss-of-function mutations in the dystrophin (DMD) gene encoding for dystrophin. Among many different approaches for DMD treatment, exon skipping using antisense oligonucleotides (ASOs) is one of the most promising methods for the restoration of dystrophin expression. ASOs are able to bind to complementary sequences of the dystrophin pre-mRNA to induce skipping of the targeted exon by modulating pre-mRNA splicing. Through the antisense therapy, ASOs can restore the open reading frame of the DMD gene and produce internally deleted, yet partially functional dystrophin protein isoforms in skeletal muscle.
Creative Biolabs is a world-renowned service provider for antisense oligonucleotides therapy development. We provide a variety of top-quality ASO products for DMD therapy. A series of stringent criteria are applied to implement quality control of antisense products in order to guarantee reliability. We are dedicated to providing the best-characterized antisense therapy service to help our customers and expedite their project application. Please feel free to contact us by e-mail for a quote and further discussion with our scientists.
Reference
- Koo, T. and Wood, M. (2013). Clinical Trials Using Antisense Oligonucleotides in Duchenne Muscular Dystrophy. Human Gene Therapy. 24(5), pp.479-488.
