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2018 ; 25
(5
): 357-364
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Tapping the RNA world for therapeutics
#MMPMID29662218
Lieberman J
Nat Struct Mol Biol
2018[May]; 25
(5
): 357-364
PMID29662218
show ga
A recent revolution in RNA biology has led to the identification of new RNA
classes with unanticipated functions, new types of RNA modifications, an
unexpected multiplicity of alternative transcripts and widespread transcription
of extragenic regions. This development in basic RNA biology has spawned a
corresponding revolution in RNA-based strategies to generate new types of
therapeutics. Here, I review RNA-based drug design and discuss barriers to
broader applications and possible ways to overcome them. Because they target
nucleic acids rather than proteins, RNA-based drugs promise to greatly extend the
domain of 'druggable' targets beyond what can be achieved with small molecules
and biologics.