Chemistry, structure and function of approved oligonucleotide therapeutics

M Egli, M Manoharan - Nucleic Acids Research, 2023 - academic.oup.com
Eighteen nucleic acid therapeutics have been approved for treatment of various diseases in
the last 25 years. Their modes of action include antisense oligonucleotides (ASOs), splice …

Gold nanoparticles for biology and medicine

DA Giljohann, DS Seferos, WL Daniel… - Spherical Nucleic …, 2020 - api.taylorfrancis.com
Gold nanoparticles (AuNPs) have a rich history in chemistry, dating back to ancient R beg
glasses f an ov or decor er 150 y ati ears ago ve purpose oman times w s. The modern here …

Nanoparticles and their applications in cell and molecular biology

EC Wang, AZ Wang - Integrative biology, 2014 - academic.oup.com
Nanoparticles can be engineered with distinctive composition, size, shape, and surface
chemistry to enable novel techniques in a wide range of biological applications. The unique …

Ultrasmall gold nanoparticles as carriers for nucleus-based gene therapy due to size-dependent nuclear entry

S Huo, S Jin, X Ma, X Xue, K Yang, A Kumar… - ACS …, 2014 - ACS Publications
The aim of this study was to determine the size-dependent penetration ability of gold
nanoparticles and the potential application of ultrasmall gold nanoparticles for intranucleus …

Oligonucleotide-modified gold nanoparticles for intracellular gene regulation

NL Rosi, DA Giljohann, CS Thaxton, AKR Lytton-Jean… - Science, 2006 - science.org
We describe the use of gold nanoparticle-oligonucleotide complexes as intracellular gene
regulation agents for the control of protein expression in cells. These oligonucleotide …

Antisense technologies: improvement through novel chemical modifications

J Kurreck - European journal of biochemistry, 2003 - Wiley Online Library
Antisense agents are valuable tools to inhibit the expression of a target gene in a sequence‐
specific manner, and may be used for functional genomics, target validation and therapeutic …

siRNAs: applications in functional genomics and potential as therapeutics

Y Dorsett, T Tuschl - Nature reviews Drug discovery, 2004 - nature.com
Molecules that can specifically silence gene expression are powerful research tools. Much
effort has been put into the development of such molecules and has resulted in the creation …

Design of antisense oligonucleotides stabilized by locked nucleic acids

J Kurreck, E Wyszko, C Gillen… - Nucleic acids …, 2002 - academic.oup.com
The design of antisense oligonucleotides containing locked nucleic acids (LNA) was
optimized and compared to intensively studied DNA oligonucleotides, phosphorothioates …

Nucleic-acid therapeutics: basic principles and recent applications

JB Opalinska, AM Gewirtz - Nature Reviews Drug Discovery, 2002 - nature.com
The sequencing of the human genome and the elucidation of many molecular pathways that
are important in disease have provided unprecedented opportunities for the development of …

Specificity of short interfering RNA determined through gene expression signatures

D Semizarov, L Frost, A Sarthy… - Proceedings of the …, 2003 - National Acad Sciences
Short interfering RNA (siRNA) is widely used for studying gene function and holds great
promise as a tool for validating drug targets and treating disease. A critical assumption in …