Our Research

ASO Therapeutic Strategies

Pre-mRNA splicing is a critical step in eukaryotic gene expression. Defects in the splicing process cause many different human diseases and mutations that alter splicing are common. We are investigating mechanisms of pre-mRNA splicing and regulation of alternative splicing and applying these insights to determine how splicing is deregulated in disease.

See how we apply these methods to:

Cystic Fibrosis

CLN3 Batten Disease

Alzheimer's Disease