Antisense Oligonucleotide Design and Evaluation of Splice-Modulating Properties Using Cell-Based Assays
Publication year
2018Publisher
[S.l.] : Springer
Series
MIMB ; 1828
In
Methods in Molecular Biology, pp. 519-530Publication type
Part of book or chapter of book

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Organization
Human Genetics
Otorhinolaryngology
Book title
Methods in Molecular Biology
Page start
p. 519
Page end
p. 530
Subject
MIMB; Radboudumc 12: Sensory disorders DCMN: Donders Center for Medical NeuroscienceAbstract
Antisense oligonucleotide (AON)-based splice modulation has been proven to hold great promise as a therapeutic strategy for a number of hereditary conditions. AONs are small modified single-stranded RNA or DNA molecules that are complementary to splice enhancer or silencer target sites. Upon pre-mRNA binding, AONs will prevent or stimulate binding of the spliceosome thereby modulating splicing events. AONs can be designed and applied for different genes and genetic disorders as the specificity depends on their nucleotide sequence. Here we provide a guideline for setting up AON-based splice-modulation experiments by describing a detailed protocol to design and evaluate AONs using a combination of in silico and in vitro analyses.
This item appears in the following Collection(s)
- Academic publications [227942]
- Faculty of Medical Sciences [86237]
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