TY  - JOUR
A1  - Schmidt, Marco F.
T1  - miRNA Targeting Drugs
BT  - the next blockbusters?
JF  - Drug Target miRNA: Methods and Protocols
N2  - Only 20 years after the discovery of small non-coding, single-stranded ribonucleic acids, so-called microRNAs (miRNAs), as post-transcriptional gene regulators, the first miRNA-targeting drug Miravirsen for the treatment of hepatitis C has been successfully tested in clinical Phase II trials. Addressing miRNAs as drug targets may enable the cure, or at least the treatment of diseases, which presently seems impossible. However, due to miRNAs’ chemical structure, generation of potential drug molecules with necessary pharmacokinetic properties is still challenging and requires a re-thinking of the drug discovery process. Therefore, this chapter highlights the potential of miRNAs as drug targets, discusses the challenges, and tries to give a complete overview of recent strategies in miRNA drug discovery.
KW  - miRNA
KW  - Drug discovery
KW  - microRNA-induced silencing complex
KW  - Antisense agents
KW  - Small-molecule miRNA modulators
KW  - Argonaute 2 protein
Y1  - 2016
SN  - 978-1-4939-6563-2
SN  - 978-1-4939-6561-8
U6  - https://doi.org/10.1007/978-1-4939-6563-2_1
SN  - 1064-3745
SN  - 1940-6029
VL  - 1517
SP  - 3
EP  - 22
PB  - Springer
CY  - New York
ER  -