Support for the discovery and development of antisense therapeutics
Oligonucleotide therapeutics, including antisense oligonucleotides (ASOs) and RNA interference (RNAi), represent a powerful modality for the treatment of many diseases, particularly genetic disorders associated with aberrant protein production. The most famous example of oligonucleotide-based intervention is in the treatment of the neuromuscular disorder spinal muscular atrophy (SMA), associated with mutations in the SMN1 gene. Treatment with an ASO induces alternate splicing of the SMN2 transcript, leading to the generation of SMN protein that functionally replaces the missing SMN1 product.
The efficacy of this approach has led to continuing interest in this therapeutic modality. Our services can support the selection of optimal ASO/RNAi targets, the analysis of oligo species, and the comprehensive characterization of oligonucleotide treatment effects.








