In Silico Design of High Efficacy Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy (DMD)

Objective

• To develop a computational framework for the treatment of genetic diseases such as DMD
• To improve patients’ life expectancy from 25 years to a normal life span using personalized medicine
• Realize personalized medicine as an optimized treatment option

Impact

• Faster, less expensive treatment options with fewer side effects
• Drug development for optimal treatment of genetic diseases

Key Milestones

• Explore importance of structural features in oligos and targets to exon skipping (designing algorithm)
• Improve the energy model to calculate binding affinity of oligos to target
• Identify influential variables in efficacy of binding oligo to target
• Improve the prediction accuracy of exon skipping efficacy of binding oligos to target
• Develop a freely available software framework for the community
• Determine economic and commercial viability