The Prospects for Antisense Therapy

Abstract

The use of short spans of nucleic acid—DNA or RNA—to disrupt the expression of disease-related genetic code is an idea potentially applicable to a vast number of illnesses, including most neoplasms. On several fronts, including hematologic malignancies, the concept has reached clinical trials—where appreciation of the in vivo hurdles is leading to new approaches for delivery of this new magic bullet.