DNA aptamers against the DUX4 protein reveal novel therapeutic implications for FSHD
Abstract
Aberrant expression of the transcription factor double homeobox protein 4 (DUX4) can lead to a number of diseases including facio-scapulo-humeral muscular dystrophy (FSHD), acute lymphoblastic leukemia, and sarcomas. Inhibition of DUX4 may represent a therapeutic strategy for these diseases. By applying Systematic Evolution of Ligands by EXponential Enrichment (SELEX), we identified aptamers against DUX4 with specific secondary structural elements conveying high affinity to DUX4 as assessed by fluorescence resonance energy transfer and fluorescence polarization techniques. Sequences analysis of these aptamers revealed the presence of two consensus DUX4 motifs in a reverse complementary fashion forming hairpins interspersed with bulge loops at distinct positions that enlarged the binding surface with the DUX4 protein, as determined by crystal structure analysis. We demonstrate that insertion of specific structural elements into transcription factor binding oligonucleotides can enhance specificity and affinity.
- Authors:
-
- Neuromuscular Research Group Department of Neurology University Hospital Basel Basel Switzerland, Neuromuscular Research Group Department of Biomedicine University Hospital Basel Basel Switzerland
- Neuromuscular Research Group Department of Neurology University Hospital Basel Basel Switzerland, Neuromuscular Research Group Department of Biomedicine University Hospital Basel Basel Switzerland, Department of Health Technology Technical University of Denmark Kgs Lyngby Denmark
- Department of Biochemistry, Molecular Biology, and Biophysics University of Minnesota Minneapolis MN USA
- Lillehei Heart Institute University of Minnesota Minneapolis MN USA, Department of Pediatrics University of Minnesota Minneapolis MN USA
- Publication Date:
- Research Org.:
- Argonne National Lab. (ANL), Argonne, IL (United States)
- Sponsoring Org.:
- USDOE; National Institutes of Health (NIH)
- OSTI Identifier:
- 1597528
- Alternate Identifier(s):
- OSTI ID: 1597529; OSTI ID: 1605447
- Grant/Contract Number:
- DE‐AC02‐06CH11357
- Resource Type:
- Published Article
- Journal Name:
- FASEB Journal
- Additional Journal Information:
- Journal Name: FASEB Journal Journal Volume: 34 Journal Issue: 3; Journal ID: ISSN 0892-6638
- Publisher:
- FASEB
- Country of Publication:
- United States
- Language:
- English
- Subject:
- 59 BASIC BIOLOGICAL SCIENCES; bulge loop; decoy; PAX7; PROP1; transcription factor
Citation Formats
Klingler, Christian, Ashley, Jon, Shi, Ke, Stiefvater, Adeline, Kyba, Michael, Sinnreich, Michael, Aihara, Hideki, and Kinter, Jochen. DNA aptamers against the DUX4 protein reveal novel therapeutic implications for FSHD. United States: N. p., 2020.
Web. doi:10.1096/fj.201902696.
Klingler, Christian, Ashley, Jon, Shi, Ke, Stiefvater, Adeline, Kyba, Michael, Sinnreich, Michael, Aihara, Hideki, & Kinter, Jochen. DNA aptamers against the DUX4 protein reveal novel therapeutic implications for FSHD. United States. https://doi.org/10.1096/fj.201902696
Klingler, Christian, Ashley, Jon, Shi, Ke, Stiefvater, Adeline, Kyba, Michael, Sinnreich, Michael, Aihara, Hideki, and Kinter, Jochen. Wed .
"DNA aptamers against the DUX4 protein reveal novel therapeutic implications for FSHD". United States. https://doi.org/10.1096/fj.201902696.
@article{osti_1597528,
title = {DNA aptamers against the DUX4 protein reveal novel therapeutic implications for FSHD},
author = {Klingler, Christian and Ashley, Jon and Shi, Ke and Stiefvater, Adeline and Kyba, Michael and Sinnreich, Michael and Aihara, Hideki and Kinter, Jochen},
abstractNote = {Aberrant expression of the transcription factor double homeobox protein 4 (DUX4) can lead to a number of diseases including facio-scapulo-humeral muscular dystrophy (FSHD), acute lymphoblastic leukemia, and sarcomas. Inhibition of DUX4 may represent a therapeutic strategy for these diseases. By applying Systematic Evolution of Ligands by EXponential Enrichment (SELEX), we identified aptamers against DUX4 with specific secondary structural elements conveying high affinity to DUX4 as assessed by fluorescence resonance energy transfer and fluorescence polarization techniques. Sequences analysis of these aptamers revealed the presence of two consensus DUX4 motifs in a reverse complementary fashion forming hairpins interspersed with bulge loops at distinct positions that enlarged the binding surface with the DUX4 protein, as determined by crystal structure analysis. We demonstrate that insertion of specific structural elements into transcription factor binding oligonucleotides can enhance specificity and affinity.},
doi = {10.1096/fj.201902696},
journal = {FASEB Journal},
number = 3,
volume = 34,
place = {United States},
year = {2020},
month = {2}
}
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