Antisense Oligonucleotide Design and Evaluation of Splice-Modulating Properties Using Cell-Based Assays

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Title:	Antisense Oligonucleotide Design and Evaluation of Splice-Modulating Properties Using Cell-Based Assays
Author(s):	Slijkerman, R.W. ; Kremer, H. ; WIjk, E. van
Publication year:	2018
Source:	Methods in Molecular Biology, vol. 1828, (2018), pp. 519-530
ISSN:	1064-3745
DOI:	https://doi.org/10.1007/978-1-4939-8651-4_34
Publication type:	Article / Letter to editor
Please use this identifier to cite or link to this item : https://hdl.handle.net/2066/196432
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Subject:	Radboudumc 12: Sensory disorders DCMN: Donders Center for Medical Neuroscience Tijdelijke code tbv inlezen publicaties Radboudumc - Alleen voor gebruik door Radboudumc
Organization:	Otorhinolaryngology Human Genetics
Journal title:	Methods in Molecular Biology
Volume:	vol. 1828
Page start:	p. 519
Page end:	p. 530
Abstract:	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.