What is Alternative Splicing?

In human DNA, approximately 20,000 genes code for hundreds of thousands of distinct proteins required for normal tissue development and function. This is possible because a single gene can translate into multiple protein variants, with distinct and sometimes opposing function, through a dynamic and heavily regulated mechanism known as alternative pre-mRNA splicing, commonly known as alternative splicing.

Alternative pre-mRNA Splicing and Disease

Alternative splicing regulates genome potential by differentially joining or skipping gene segments at alternative splice sites. The resulting proteome diversification is a fundamental physiological aspect of tissue fate and function. Conversely, dysregulated alternative splicing can be a root cause of developmental disorders, tissue degeneration and cancer.

What We Do

Biosplice Therapeutics has produced fresh biological insights and unique chemical equity that delivers therapeutic modulation of alternative splicing by targeting the CLK/DYRK family kinases. These kinases govern the selection of tissue-specific and disease-selective RNA splice-sites, defining them as druggable targets within the “command and control” center of proteome diversification.