RNA: THE BASICS
Humans have about 22,000 genes, which contain the blueprints for producing proteins that perform essential functions in the body.
Proteins are molecular workhorses involved in almost every function in our bodies, and defective proteins often result in disease. More specifically, some diseases may be caused by the over-production of one or more proteins, while other diseases are caused by protein deficiencies.
Proteins are produced in cells, where genes in the DNA are "transcribed" into RNA templates, which are then processed and "translated" into proteins by the cellular machinery.
EXPLORE PROTEIN SYNTHESIS
Our RNA-targeted therapeutics direct the cellular machinery involved in making proteins. These drugs can be designed to increase or decrease the production of a protein involved in a disease.
By working at the genetic level, RNA-targeted therapeutics are powerful tools with the potential to address diseases that otherwise could not be treated with traditional small molecule or biologic drugs.