Chemical modification of nucleic acids as a key technology for the development of RNA-based therapeutics

Grünweller, A.; Hartmann, R.K.

Die Pharmazie 71(1): 8-16

2016


ISSN/ISBN: 0031-7144
PMID: 26867347
Document Number: 690153
RNA-based effector molecules (nucleic acid effectors) are important tools in molecular medicine because they offer a strategy to address therapeutically interesting targets that are not "druggable" with classic small molecule inhibitors. However, for in vivo applications, RNA-based effectors require specific chemical modifications to improve their stability and pharmacokinetic properties, as well as to minimize toxic and unspecific off-target effects.

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