학술논문

Nucleic Acid Therapeutics for Hematologic Malignancies—Theoretical Considerations.
Document Type
Article
Source
Annals of the New York Academy of Sciences. 2006, Vol. 1082 Issue 1, p124-136. 13p. 1 Color Photograph, 3 Diagrams, 1 Chart, 2 Graphs.
Subject
*RNA
*THERAPEUTICS
*BLOOD diseases
*HEMATOLOGIC agents
*SMALL interfering RNA
Language
ISSN
0077-8923
Abstract
Our work is motivated by the belief that RNA targeted gene silencing agents can be developed into effective drugs for treating hematologic malignancies. In many experimental systems, antisense nucleic acids of various composition, including antisense oligodeoxynucleotides (AS ODNs) and short interfering RNA (siRNA), have been shown to perturb gene expression in a sequence specific manner. Nevertheless, our clinical experience, and those of others, have led us to conclude that the antisense nucleic acids (ASNAs) we, and others, employ need to be optimized with regard to intracellular delivery, targeting, chemical composition, and efficiency of mRNA destruction. We have hypothesized that addressing these critical issues will lead to the development of practical and effective nucleic acid drugs. An overview of our recent work which seeks to addresses these core issues is contained within this review. [ABSTRACT FROM AUTHOR]