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Engineering Artificial Factors to Specifically Manipulate Alternative Splicing in Human Cells
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In Vitro Modulation of Endogenous Alternative Splicing Using Splice-Switching Antisense Oligonucleotides
Jeong Eun Park1, Luca Cartegni2
1Susan Lehman Cullman Laboratory for Cancer Research, Ernest Mario School of Pharmacy, Rutgers, The State University of New Jersey, Piscataway, NJ, USA.
Methods in Molecular Biology (Clifton, N.J.)
|August 3, 2017
Summary
Antisense oligonucleotides offer a powerful method to control gene expression by regulating alternative splicing. These splice-switching compounds, like phosphorodiamidate morpholinos (PMOs), enable precise modulation of RNA processing for therapeutic applications.
Area of Science:
- Molecular Biology
- Genetics
- RNA Biology
Background:
- Alternative splicing is a key mechanism for gene expression regulation.
- Dysregulation of alternative splicing is linked to various diseases.
- Antisense technology provides a means to target RNA processing.
Purpose of the Study:
- To describe methods for inducing and analyzing RNA processing modulation.
- To highlight the utility of splice-switching antisense oligonucleotides.
- To demonstrate control over gene expression via alternative splicing.
Main Methods:
- Utilizing modified splice-switching antisense oligonucleotides.
- Employing phosphorodiamidate morpholino (PMO) compounds.
- In vitro and in vivo experimental approaches.
Main Results:
- Successful modulation of endogenous alternative splicing patterns.
- Correction of cryptic or aberrant splicing events.
- Reduction of gene expression through nonsense-mediated mRNA decay induction.
- Activation of intronic polyadenylation.
Conclusions:
- Antisense-mediated splicing interference is a versatile molecular tool.
- This approach allows for precise control of RNA processing.
- Applications include therapeutic correction of splicing defects and gene expression modulation.
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