Splice-switching antisense oligonucleotides as therapeutic drugs

MA Havens, ML Hastings - Nucleic acids research, 2016 - academic.oup.com
Splice-switching oligonucleotides (SSOs) are short, synthetic, antisense, modified nucleic
acids that base-pair with a pre-mRNA and disrupt the normal splicing repertoire of the …

RNA therapeutics: beyond RNA interference and antisense oligonucleotides

R Kole, AR Krainer, S Altman - Nature reviews Drug discovery, 2012 - nature.com
Here, we discuss three RNA-based therapeutic technologies exploiting various
oligonucleotides that bind to RNA by base pairing in a sequence-specific manner yet have …

Targeting eosinophils in allergy, inflammation and beyond

PC Fulkerson, ME Rothenberg - Nature reviews Drug discovery, 2013 - nature.com
Eosinophils can regulate local immune and inflammatory responses, and their accumulation
in the blood and tissue is associated with several inflammatory and infectious diseases …

Alternative splicing: a new cause and potential therapeutic target in autoimmune disease

P Ren, L Lu, S Cai, J Chen, W Lin, F Han - Frontiers in Immunology, 2021 - frontiersin.org
Alternative splicing (AS) is a complex coordinated transcriptional regulatory mechanism. It
affects nearly 95% of all protein-coding genes and occurs in nearly all human organs …

Locked vs. unlocked nucleic acids (LNA vs. UNA): contrasting structures work towards common therapeutic goals

MA Campbell, J Wengel - Chemical Society Reviews, 2011 - pubs.rsc.org
Oligonucleotide chemistry has been developed greatly over the past three decades, with
many advances in increasing nuclease resistance, enhancing duplex stability and assisting …

Targeting RNA splicing for disease therapy

MA Havens, DM Duelli… - Wiley Interdisciplinary …, 2013 - Wiley Online Library
Splicing of pre‐messenger RNA into mature messenger RNA is an essential step for the
expression of most genes in higher eukaryotes. Defects in this process typically affect …

A chemical view of oligonucleotides for exon skipping and related drug applications

P Järver, L O'Donovan, MJ Gait - Nucleic Acid Therapeutics, 2014 - liebertpub.com
Exon skipping is one of a number of ''steric blocking''applications of oligonucleotides (ONs)
and their analogues that in recent years have undergone a renaissance and which have led …

Anti-tumor activity of splice-switching oligonucleotides

JA Bauman, SD Li, A Yang, L Huang… - Nucleic acids …, 2010 - academic.oup.com
Alternative splicing has emerged as an important target for molecular therapies. Splice-
switching oligonucleotides (SSOs) modulate alternative splicing by hybridizing to pre-mRNA …

Therapeutic potential of splice-switching oligonucleotides

J Bauman, N Jearawiriyapaisarn, R Kole - Oligonucleotides, 2009 - liebertpub.com
Alternative splicing enables a single pre-messenger RNA transcript to yield multiple protein
isoforms, making it a major contributor to the diversity of the proteome. While this process is …

[HTML][HTML] Protein isoform-centric therapeutics: expanding targets and increasing specificity

P Kjer-Hansen, TG Phan, RJ Weatheritt - Nature Reviews Drug …, 2024 - nature.com
Most protein-coding genes produce multiple protein isoforms; however, these isoforms are
commonly neglected in drug discovery. The expression of protein isoforms can be specific to …