Antisense Therapy

Antisense drugs are small, chemically modified strands of DNA that are engineered in a sequence that is exactly opposite ("anti") to the coding ("sense") sequence of messenger RNA (mRNA). When an antisense drug binds to the mRNA, a duplex is formed. The duplex recruits an enzyme that degrades mRNA, thereby inhibiting the production of the intended protein. In preclinical studies, BCL-2 antisense therapy has confirmed downregulation of Bcl-2 (a protein found in the mitochondrial membrane that regulates the release of cytochrome c), demonstrated a synergistic effect between antisense and chemotherapeutic agents, and has shown a significant tumor reduction and increased survival.