Polyornithine-based polyplexes to boost effective gene silencing in CNS disorders
Gene silencing therapies have successfully suppressed the translation of target proteins, a strategy that holds great promise for the treatment of central nervous system (CNS) disorders. Advances in the current knowledge on multimolecular delivery vehicles are concentrated on overcoming the difficul...
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| Veröffentlicht in: | Nanoscale 2020-03, Vol.12 (11), p.6285-6299 |
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| creator | Conejos-Sánchez, I Gallon, E Niño-Pariente, A Smith, J. A De la Fuente, A. G Di Canio, L Pluchino, S Franklin, R. J. M Vicent, M. J |
| description | Gene silencing therapies have successfully suppressed the translation of target proteins, a strategy that holds great promise for the treatment of central nervous system (CNS) disorders. Advances in the current knowledge on multimolecular delivery vehicles are concentrated on overcoming the difficulties in delivery of small interfering (si)RNA to target tissues, which include anatomical accessibility, slow diffusion, safety concerns, and the requirement for specific cell uptake within the unique environment of the CNS. The present work addressed these challenges through the implementation of polyornithine derivatives in the construction of polyplexes used as non-viral siRNA delivery vectors. Physicochemical and biological characterization revealed biodegradability and biocompatibility of our polyornithine-based system and the ability to silence gene expression in primary oligodendrocyte progenitor cells (OPCs) effectively. In summary, the well-defined properties and neurological compatibility of this polypeptide-based platform highlight its potential utility in the treatment of CNS disorders.
Novel biodegradable and biocompatible polyornithine derivatives as non-viral vectors for siRNA exhibit effectively silence gene expression in primary neural cells. |
| doi_str_mv | 10.1039/c9nr06187h |
| format | Article |
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Novel biodegradable and biocompatible polyornithine derivatives as non-viral vectors for siRNA exhibit effectively silence gene expression in primary neural cells.</description><subject>Biocompatibility</subject><subject>Biodegradability</subject><subject>Cell Line, Tumor</subject><subject>Cells (biology)</subject><subject>Central nervous system</subject><subject>Central Nervous System Diseases - genetics</subject><subject>Central Nervous System Diseases - metabolism</subject><subject>Central Nervous System Diseases - pathology</subject><subject>Central Nervous System Diseases - therapy</subject><subject>Diffusion rate</subject><subject>Disorders</subject><subject>Gene expression</subject><subject>Gene Silencing</subject><subject>Humans</subject><subject>Oligodendroglia - metabolism</subject><subject>Oligodendroglia - pathology</subject><subject>Peptides - chemistry</subject><subject>Peptides - pharmacology</subject><subject>Polypeptides</subject><subject>RNA, Small Interfering - chemistry</subject><subject>RNA, Small Interfering - genetics</subject><subject>RNA, Small Interfering - pharmacology</subject><subject>Stem Cells - metabolism</subject><subject>Stem Cells - pathology</subject><issn>2040-3364</issn><issn>2040-3372</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2020</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNp9kc1LAzEQxYMoVqsX70rEiwir-epuc5SiVij1-7xssrNtZJusyVbsf2-0tYIHmcMM834MjzcIHVByTgmXF1paT1Laz6YbaIcRQRLOM7a5nlPRQbshvBKSSp7ybdThtC9IRrMd9HDv6oXz1rRTYyFRRYASN3HX1PABAbcOK-dCi6GqQLfmHfAELOBgarDa2Ak2Fg_GT7g0wfkSfNhDW1VRB9hf9S56ub56HgyT0d3N7eBylOgeYW0C_WiF6IpIzbhSNOVUZVproooCVKFBUah0TwjCKWcFAUilJCmTjIIse8C76HR5t_HubQ6hzWcmaKjrwoKbh5xxlvFYVET05A_66ubeRneRyvqZEILSSJ0tKe1dCB6qvPFmVvhFTkn-FXQ-kOPH76CHET5anZyrGZRr9CfZCBwuAR_0Wv39VNSP_9Pzpqz4J3u-jhg</recordid><startdate>20200321</startdate><enddate>20200321</enddate><creator>Conejos-Sánchez, I</creator><creator>Gallon, E</creator><creator>Niño-Pariente, A</creator><creator>Smith, J. 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| subjects | Biocompatibility Biodegradability Cell Line, Tumor Cells (biology) Central nervous system Central Nervous System Diseases - genetics Central Nervous System Diseases - metabolism Central Nervous System Diseases - pathology Central Nervous System Diseases - therapy Diffusion rate Disorders Gene expression Gene Silencing Humans Oligodendroglia - metabolism Oligodendroglia - pathology Peptides - chemistry Peptides - pharmacology Polypeptides RNA, Small Interfering - chemistry RNA, Small Interfering - genetics RNA, Small Interfering - pharmacology Stem Cells - metabolism Stem Cells - pathology |
| title | Polyornithine-based polyplexes to boost effective gene silencing in CNS disorders |
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