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.