Antisense and Sensibility in RNA Therapeutics

These days RNA interference seems to be everywhere.

Written byLaura Lane
| 6 min read

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Courtesy of Antisense Therapeutics Ltd.

Antisense strategies use polymeric nucleic acids or nucleic acid analogs to bind to and silence specific messenger RNAs. The silencing can be caused either by physically blocking the translational machinery or by RNA degradation via RNAse H. cleotides for research applications.)

These days RNA interference seems to be everywhere. A bonafide hit in research labs worldwide, the sequence-specific gene-silencing approach is now making inroads in drug-development circles. But RNAi is not the first targeted nucleic acid-based approach to garner both accolades and pharmaceutical dollars.

For at least 30 years scientists and drug developers bent on a sorely needed new class of therapeutics have been studying antisense RNA. The concept is deceptively simple: Binding of an oligonucleotide complementary to a specific mRNA transcript – in other words, an antisense molecule – to that transcript either blocks translation of, or stimulates degradation of, the mRNA.

Implementation, however, ...

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