New modalities for challenging targets in drug discovery

E Valeur, SM Guéret, H Adihou… - Angewandte Chemie …, 2017 - Wiley Online Library
Our ever‐increasing understanding of biological systems is providing a range of exciting
novel biological targets, whose modulation may enable novel therapeutic options for many …

[HTML][HTML] Designing chemically modified oligonucleotides for targeted gene silencing

GF Deleavey, MJ Damha - Chemistry & biology, 2012 - cell.com
Oligonucleotides (ONs), and their chemically modified mimics, are now routinely used in the
laboratory as a means to control the expression of fundamentally interesting or …

[HTML][HTML] Expanding RNAi therapeutics to extrahepatic tissues with lipophilic conjugates

KM Brown, JK Nair, MM Janas… - Nature …, 2022 - nature.com
Therapeutics based on short interfering RNAs (siRNAs) delivered to hepatocytes have been
approved, but new delivery solutions are needed to target additional organs. Here we show …

RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform

CF Bennett, EE Swayze - Annual review of pharmacology and …, 2010 - annualreviews.org
Dramatic advances in understanding of the roles RNA plays in normal health and disease
have greatly expanded over the past 10 years and have made it clear that scientists are only …

[HTML][HTML] Locked nucleic acid: modality, diversity, and drug discovery

PH Hagedorn, R Persson, ED Funder, N Albæk… - Drug Discovery …, 2018 - Elsevier
Highlights•Locked nucleic acid-modified antisense oligonucleotides (LNAs) bind to and
modulate RNA.•The high binding affinity of LNAs significantly improves potency.•Structural …

[图书][B] Nucleic acids in chemistry and biology

GM Blackburn - 2006 - books.google.com
The structure, function and reactions of nucleic acids are central to molecular biology and
are crucial for the understanding of complex biological processes involved. Revised and …

Chemical Modification of siRNAs for In Vivo Use

MA Behlke - Oligonucleotides, 2008 - liebertpub.com
Well over a hundred reports have been published describing use of synthetic small-
interfering RNAs (siRNAs) in animals. The majority of these reports employed unmodified …

Peptidic tools applied to redirect alternative splicing events

MM Nancy, RM Nora, MC Rebeca - Peptides, 2015 - Elsevier
Peptides are versatile and attractive biomolecules that can be applied to modulate genetic
mechanisms like alternative splicing. In this process, a single transcript yields different …

[HTML][HTML] Progress towards in vivo use of siRNAs

MA Behlke - Molecular Therapy, 2006 - cell.com
RNA interference (RNAi) has become the method of choice to suppress gene expression in
vitro. It is also emerging as a powerful tool for in vivo research with over 90 studies …

Overcoming the challenges of tissue delivery for oligonucleotide therapeutics

T Gökirmak, M Nikan, S Wiechmann, TP Prakash… - Trends in …, 2021 - cell.com
Synthetic therapeutic oligonucleotides (STO) represent the third bonafide platform for drug
discovery in the pharmaceutical industry after small molecule and protein therapeutics. So …