Abstract
In This Issue...
The field of nucleic acid-based therapeutics ranges from gene therapy
technologies for replacing defective or missing genes to gene-blocking or
gene-silencing technologies for preventing the expression of deleterious
genes. RNA-based therapeutics are a relatively new addition to the field, and
we focus on three such technologies in this report: antisense
oligonucleotides, RNA interference, and microRNAs. These three technologies
are particularly dynamic; antisense, for example, has suffered a series of
negative clinical trial results that may have stalled its progress, and RNA
interference has been hailed as a revolutionary breakthrough and has generated
interest from more than 100 companies. In this report, we describe these three
RNA-based technologies, review recent progress in RNA-based drug discovery and
development, and profile the activities of companies in the field.
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