Respirable Antisense Oligonucleotides: A New, Third Drug Class Targeting Respiratory Disease

Jonathan Nyce


Curr Opin Allergy Clin Immunol. 2002;2(6) 

In This Article


Respirable antisense oligonucleotides represent a powerful new class of respiratory drugs with the potential to offer certain advantages over current respiratory therapeutics. They also represent potentially powerful tools that can be used, in appropriate animal models of respiratory disease, to discover the best of the next generation of respiratory targets. The use of mismatch controls (two mismatches is the optimal number) and not scrambled or sense controls, which can contain sequence motifs with possible biological function, the establishment of a dose-response curve, and assessing effects upon closely related genes to establish specificity, are key components of establishing that an antisense mechanism is at work. The potential of this new technology has been only minutely tapped to date, but is likely to be exploited more fully in the coming years.


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