Translational development of splice-modifying antisense oligomers
Introduction: Antisense nucleic acid analogues can interact with pre-mRNA motifs and
influence exon or splice site selection and thereby alter gene expression. Design of …
influence exon or splice site selection and thereby alter gene expression. Design of …
Precision medicine through antisense oligonucleotide-mediated exon skipping
Clinical implementation of two recently approved antisense RNA therapeutics–Exondys51®
to treat Duchenne muscular dystrophy (Duchenne MD) and Spinraza® as a treatment for …
to treat Duchenne muscular dystrophy (Duchenne MD) and Spinraza® as a treatment for …
Antisense-mediated modulation of splicing: therapeutic implications for Duchenne muscular dystrophy
A Aartsma-Rus - RNA biology, 2010 - Taylor & Francis
While disruption of alternative splicing underlies many diseases, modulation of splicing
using antisense oligonucleotides (AONs) can have therapeutic implications. The most …
using antisense oligonucleotides (AONs) can have therapeutic implications. The most …
Splicing modulation therapy in the treatment of genetic diseases
V Arechavala-Gomeza, B Khoo… - The application of …, 2014 - Taylor & Francis
Antisense-mediated splicing modulation is a tool that can be exploited in several ways to
provide a potential therapy for rare genetic diseases. This approach is currently being tested …
provide a potential therapy for rare genetic diseases. This approach is currently being tested …
Development of therapeutic splice-switching oligonucleotides
P Disterer, A Kryczka, Y Liu, YE Badi, JJ Wong… - Human gene …, 2014 - liebertpub.com
Synthetic splice-switching oligonucleotides (SSOs) target nuclear pre-mRNA molecules to
change exon splicing and generate an alternative protein isoform. Clinical trials with two …
change exon splicing and generate an alternative protein isoform. Clinical trials with two …
Delivery is key: lessons learnt from developing splice‐switching antisense therapies
The use of splice‐switching antisense therapy is highly promising, with a wealth of pre‐
clinical data and numerous clinical trials ongoing. Nevertheless, its potential to treat a variety …
clinical data and numerous clinical trials ongoing. Nevertheless, its potential to treat a variety …
[HTML][HTML] Systematic approach to developing splice modulating antisense oligonucleotides
The process of pre-mRNA splicing is a common and fundamental step in the expression of
most human genes. Alternative splicing, whereby different splice motifs and sites are …
most human genes. Alternative splicing, whereby different splice motifs and sites are …
Tips to design effective splice-switching antisense oligonucleotides for exon skipping and exon inclusion
R Maruyama, T Yokota - Exon Skipping and Inclusion Therapies: Methods …, 2018 - Springer
Antisense-mediated exon skipping and exon inclusion have proven to be powerful tools for
treating neuromuscular diseases. The approval of Exondys 51 (eteplirsen) and Spinraza …
treating neuromuscular diseases. The approval of Exondys 51 (eteplirsen) and Spinraza …
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 …
acids that base-pair with a pre-mRNA and disrupt the normal splicing repertoire of the …
Targeting RNA to treat neuromuscular disease
F Muntoni, MJA Wood - Nature reviews Drug discovery, 2011 - nature.com
The development of effective therapies for neuromuscular disorders such as Duchenne
muscular dystrophy (DMD) is hampered by considerable challenges: skeletal muscle is the …
muscular dystrophy (DMD) is hampered by considerable challenges: skeletal muscle is the …