Bioactive Aliphatic Polycarbonates Carrying Guanidinium Functions : An Innovative Approach for Myotonic Dystrophy Type 1 Therapy
Copyright © 2019 American Chemical Society..
Dystrophia myotonica type 1 (DM1) results from nuclear sequestration of splicing factors by a messenger RNA (mRNA) harboring a large (CUG) n repeat array transcribed from the causal (CTG) n DNA amplification. Several compounds were previously shown to bind the (CUG) n RNA and release the splicing factors. We now investigated for the first time the interaction of an aliphatic polycarbonate carrying guanidinium functions to DM1 DNA/RNA model probes by affinity capillary electrophoresis. The apparent association constants (K a) were in the range described for reference compounds such as pentamidine. Further macromolecular engineering could improve association specificity. The polymer presented no toxicity in cell culture at concentrations of 1.6-100.0 μg/mL as evaluated both by MTT and real-time monitoring xCELLigence method. These promising results may lay the foundation for a new branch of potential therapeutic agents for DM1.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2019 |
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| Erschienen: |
2019 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:4 |
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| Enthalten in: |
ACS omega - 4(2019), 19 vom: 05. Nov., Seite 18126-18135 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Baroni, Alexandra [VerfasserIn] |
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| Anmerkungen: |
Date Revised 29.09.2020 published: Electronic-eCollection Citation Status PubMed-not-MEDLINE |
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| doi: |
10.1021/acsomega.9b02034 |
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| funding: |
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| PPN (Katalog-ID): |
NLM303240938 |
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| 520 | |a Dystrophia myotonica type 1 (DM1) results from nuclear sequestration of splicing factors by a messenger RNA (mRNA) harboring a large (CUG) n repeat array transcribed from the causal (CTG) n DNA amplification. Several compounds were previously shown to bind the (CUG) n RNA and release the splicing factors. We now investigated for the first time the interaction of an aliphatic polycarbonate carrying guanidinium functions to DM1 DNA/RNA model probes by affinity capillary electrophoresis. The apparent association constants (K a) were in the range described for reference compounds such as pentamidine. Further macromolecular engineering could improve association specificity. The polymer presented no toxicity in cell culture at concentrations of 1.6-100.0 μg/mL as evaluated both by MTT and real-time monitoring xCELLigence method. These promising results may lay the foundation for a new branch of potential therapeutic agents for DM1 | ||
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| 700 | 1 | |a Delbosc, Nicolas |e verfasserin |4 aut | |
| 700 | 1 | |a Wells, Mathilde |e verfasserin |4 aut | |
| 700 | 1 | |a Verdy, Laetitia |e verfasserin |4 aut | |
| 700 | 1 | |a Ansseau, Eugénie |e verfasserin |4 aut | |
| 700 | 1 | |a Vanden Eynde, Jean Jacques |e verfasserin |4 aut | |
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