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Antisense Therapy

Denise M. Lapka
Paula J. Franson
CJON 2003, 7(4), 441-443 DOI: 10.1188/03.CJON.441-443

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.

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