Side chain modified peptide nucleic acids (PNA) for knock-down of <it>six3</it> in medaka embryos
<p>Abstract</p> <p>Background</p> <p>Synthetic antisense molecules have an enormous potential for therapeutic applications in humans. The major aim of such strategies is to specifically interfere with gene function, thus modulating cellular pathways according to the the...
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| Format: | Article |
| Language: | English |
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BMC
2012-08-01
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| Series: | BMC Biotechnology |
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| Online Access: | http://www.biomedcentral.com/1472-6750/12/50 |
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| author | Dorn Sebastian Aghaallaei Narges Jung Gerlinde Bajoghli Baubak Werner Birgit Bock Holger Lindhorst Thomas Czerny Thomas |
| author_facet | Dorn Sebastian Aghaallaei Narges Jung Gerlinde Bajoghli Baubak Werner Birgit Bock Holger Lindhorst Thomas Czerny Thomas |
| author_sort | Dorn Sebastian |
| collection | DOAJ |
| description | <p>Abstract</p> <p>Background</p> <p>Synthetic antisense molecules have an enormous potential for therapeutic applications in humans. The major aim of such strategies is to specifically interfere with gene function, thus modulating cellular pathways according to the therapeutic demands. Among the molecules which can block mRNA function in a sequence specific manner are peptide nucleic acids (PNA). They are highly stable and efficiently and selectively interact with RNA. However, some properties of non-modified aminoethyl glycine PNAs (aegPNA) hamper their <it>in vivo</it> applications.</p> <p>Results</p> <p>We generated new backbone modifications of PNAs, which exhibit more hydrophilic properties. When we examined the activity and specificity of these novel phosphonic ester PNAs (pePNA) molecules in medaka (<it>Oryzias latipes</it>) embryos, high solubility and selective binding to mRNA was observed. In particular, mixing of the novel components with aegPNA components resulted in mixed PNAs with superior properties. Injection of mixed PNAs directed against the medaka <it>six3</it> gene, which is important for eye and brain development, resulted in specific <it>six3</it> phenotypes.</p> <p>Conclusions</p> <p>PNAs are well established as powerful antisense molecules. Modification of the backbone with phosphonic ester side chains further improves their properties and allows the efficient knock down of a single gene in fish embryos.</p> |
| first_indexed | 2024-12-11T14:51:27Z |
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| id | doaj.art-e3c5cf4cd8f54e9497a5942d42bef81a |
| institution | Directory Open Access Journal |
| issn | 1472-6750 |
| language | English |
| last_indexed | 2024-12-11T14:51:27Z |
| publishDate | 2012-08-01 |
| publisher | BMC |
| record_format | Article |
| series | BMC Biotechnology |
| spelling | doaj.art-e3c5cf4cd8f54e9497a5942d42bef81a2022-12-22T01:01:28ZengBMCBMC Biotechnology1472-67502012-08-011215010.1186/1472-6750-12-50Side chain modified peptide nucleic acids (PNA) for knock-down of <it>six3</it> in medaka embryosDorn SebastianAghaallaei NargesJung GerlindeBajoghli BaubakWerner BirgitBock HolgerLindhorst ThomasCzerny Thomas<p>Abstract</p> <p>Background</p> <p>Synthetic antisense molecules have an enormous potential for therapeutic applications in humans. The major aim of such strategies is to specifically interfere with gene function, thus modulating cellular pathways according to the therapeutic demands. Among the molecules which can block mRNA function in a sequence specific manner are peptide nucleic acids (PNA). They are highly stable and efficiently and selectively interact with RNA. However, some properties of non-modified aminoethyl glycine PNAs (aegPNA) hamper their <it>in vivo</it> applications.</p> <p>Results</p> <p>We generated new backbone modifications of PNAs, which exhibit more hydrophilic properties. When we examined the activity and specificity of these novel phosphonic ester PNAs (pePNA) molecules in medaka (<it>Oryzias latipes</it>) embryos, high solubility and selective binding to mRNA was observed. In particular, mixing of the novel components with aegPNA components resulted in mixed PNAs with superior properties. Injection of mixed PNAs directed against the medaka <it>six3</it> gene, which is important for eye and brain development, resulted in specific <it>six3</it> phenotypes.</p> <p>Conclusions</p> <p>PNAs are well established as powerful antisense molecules. Modification of the backbone with phosphonic ester side chains further improves their properties and allows the efficient knock down of a single gene in fish embryos.</p>http://www.biomedcentral.com/1472-6750/12/50PNAKnock downMedakaSix3 |
| spellingShingle | Dorn Sebastian Aghaallaei Narges Jung Gerlinde Bajoghli Baubak Werner Birgit Bock Holger Lindhorst Thomas Czerny Thomas Side chain modified peptide nucleic acids (PNA) for knock-down of <it>six3</it> in medaka embryos BMC Biotechnology PNA Knock down Medaka Six3 |
| title | Side chain modified peptide nucleic acids (PNA) for knock-down of <it>six3</it> in medaka embryos |
| title_full | Side chain modified peptide nucleic acids (PNA) for knock-down of <it>six3</it> in medaka embryos |
| title_fullStr | Side chain modified peptide nucleic acids (PNA) for knock-down of <it>six3</it> in medaka embryos |
| title_full_unstemmed | Side chain modified peptide nucleic acids (PNA) for knock-down of <it>six3</it> in medaka embryos |
| title_short | Side chain modified peptide nucleic acids (PNA) for knock-down of <it>six3</it> in medaka embryos |
| title_sort | side chain modified peptide nucleic acids pna for knock down of it six3 it in medaka embryos |
| topic | PNA Knock down Medaka Six3 |
| url | http://www.biomedcentral.com/1472-6750/12/50 |
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