In the last decade, the field of oligonucleotide therapeutics has matured, with the regulatory
approval of several single-stranded and double-stranded RNA drugs. In this Perspective, I …

Abstract Positron Emission Tomography (PET) is an important part of the medical imaging
field which is continually exploring novel biological targets as exemplified by PET imaging of …

Antisense oligonucleotides (ASOs) are short, single-stranded nucleic acid molecules that
alter gene expression. However, their transport into appropriate cellular compartments is a …

Selection-based screening of large DNA-encoded libraries of drug-like small molecules is a
validated method to identify bioactive compounds. Among the chemical space of bioactive …

Erythropoietic protoporphyria (EPP) is a rare genetic disease in which patients experience
acute phototoxic reactions after sunlight exposure. It is caused by a deficiency in …

Micro-RNA (miRNAs) are short non-coding RNAs of about 18–20 nucleotides in length and
are implicated in many cellular processes including proliferation, development …

When used as inhibitors of gene expression in vivo, oligonucleotides require modification of
their structures to boost their binding affinity for complementary target RNAs. To date …

Functionalized oligoribonucleotides are essential tools in RNA chemical biology. Various
synthetic routes have been developed over recent years to conjugate functional groups to …

The discovery of the RNA interference (RNAi) pathway in the late 1990s–early 2000s 1
brought revitalizing optimism to the field of therapeutic gene silencing, previously dominated …

Chemical modifications to therapeutic nucleic acids are used to modulate properties such as
nuclease resistance, target engagement, enzymatic activity, and cell uptake. Therapeutic …