Therapeutic potential of antisense nucleic acid molecules
JB Opalinska, AM Gewirtz - Science's STKE, 2003 - science.org
JB Opalinska, AM Gewirtz
Science's STKE, 2003•science.orgElucidation of many disease-related signal transduction and gene expression pathways has
provided unparalleled opportunities for the development of targeted therapeutics. The types
of molecules in development are increasingly varied and include small-molecule enzyme
inhibitors, humanized antibodies to cell surface receptors, and antisense nucleic acids for
silencing the expression of specific genes. This Perspective reviews the basis for various
antisense strategies for modulating gene expression, including RNA interference, and …
provided unparalleled opportunities for the development of targeted therapeutics. The types
of molecules in development are increasingly varied and include small-molecule enzyme
inhibitors, humanized antibodies to cell surface receptors, and antisense nucleic acids for
silencing the expression of specific genes. This Perspective reviews the basis for various
antisense strategies for modulating gene expression, including RNA interference, and …
Elucidation of many disease-related signal transduction and gene expression pathways has provided unparalleled opportunities for the development of targeted therapeutics. The types of molecules in development are increasingly varied and include small-molecule enzyme inhibitors, humanized antibodies to cell surface receptors, and antisense nucleic acids for silencing the expression of specific genes. This Perspective reviews the basis for various antisense strategies for modulating gene expression, including RNA interference, and discusses the prospects for their clinical use.
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