Design of synthetic materials for intracellular delivery of RNAs: From siRNA-mediated gene silencing to CRISPR/Cas gene editing
Ribonucleic acids (RNAs) possess great therapeutic potential and can be used to treat a variety of diseases. The unique biophysical properties of RNAs, such as high molecular weight, negative charge, hydrophilicity, low stability, and potential immunogenicity, require chemical modification and devel...
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| Veröffentlicht in: | Nano research 2018-10, Vol.11 (10), p.5310-5337 |
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| creator | Miller, Jason B. Siegwart, Daniel J. |
| description | Ribonucleic acids (RNAs) possess great therapeutic potential and can be used to treat a variety of diseases. The unique biophysical properties of RNAs, such as high molecular weight, negative charge, hydrophilicity, low stability, and potential immunogenicity, require chemical modification and development of carriers to enable intracellular delivery of RNAs for clinical use. A variety of nanomaterials have been developed for the effective
in vivo
delivery of short/ small RNAs, messenger RNAs, and RNAs required for gene editing technologies including clustered regularly interspaced palindromic repeat (CRISPR)/Cas. This review outlines the challenges of delivering RNA therapeutics, explores the chemical synthesis of RNA modifications and carriers, and describes the efforts to design nanomaterials that can be used for a variety of clinical indications. |
| doi_str_mv | 10.1007/s12274-018-2099-4 |
| format | Article |
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in vivo
delivery of short/ small RNAs, messenger RNAs, and RNAs required for gene editing technologies including clustered regularly interspaced palindromic repeat (CRISPR)/Cas. This review outlines the challenges of delivering RNA therapeutics, explores the chemical synthesis of RNA modifications and carriers, and describes the efforts to design nanomaterials that can be used for a variety of clinical indications.</description><identifier>ISSN: 1998-0124</identifier><identifier>EISSN: 1998-0000</identifier><identifier>DOI: 10.1007/s12274-018-2099-4</identifier><language>eng</language><publisher>Beijing: Tsinghua University Press</publisher><subject>Atomic/Molecular Structure and Spectra ; Biomedicine ; Biotechnology ; Cellular apoptosis susceptibility protein ; Chemical modification ; Chemical synthesis ; Chemistry and Materials Science ; Condensed Matter Physics ; CRISPR ; Design modifications ; Gene silencing ; Genetic modification ; Genome editing ; Immunogenicity ; Intracellular ; Materials Science ; Molecular weight ; Nanomaterials ; Nanotechnology ; Organic chemistry ; Review Article ; Ribonucleic acid ; RNA ; siRNA ; Transcription</subject><ispartof>Nano research, 2018-10, Vol.11 (10), p.5310-5337</ispartof><rights>Tsinghua University Press and Springer-Verlag GmbH Germany, part of Springer Nature 2018</rights><rights>Nano Research is a copyright of Springer, (2018). All Rights Reserved.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c430t-c882adb8113fb8c771b356d93a7ce9f186e25785c683b51a10ba550a88e4f7953</citedby><cites>FETCH-LOGICAL-c430t-c882adb8113fb8c771b356d93a7ce9f186e25785c683b51a10ba550a88e4f7953</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://link.springer.com/content/pdf/10.1007/s12274-018-2099-4$$EPDF$$P50$$Gspringer$$H</linktopdf><linktohtml>$$Uhttps://link.springer.com/10.1007/s12274-018-2099-4$$EHTML$$P50$$Gspringer$$H</linktohtml><link.rule.ids>314,780,784,27924,27925,41488,42557,51319</link.rule.ids></links><search><creatorcontrib>Miller, Jason B.</creatorcontrib><creatorcontrib>Siegwart, Daniel J.</creatorcontrib><title>Design of synthetic materials for intracellular delivery of RNAs: From siRNA-mediated gene silencing to CRISPR/Cas gene editing</title><title>Nano research</title><addtitle>Nano Res</addtitle><description>Ribonucleic acids (RNAs) possess great therapeutic potential and can be used to treat a variety of diseases. The unique biophysical properties of RNAs, such as high molecular weight, negative charge, hydrophilicity, low stability, and potential immunogenicity, require chemical modification and development of carriers to enable intracellular delivery of RNAs for clinical use. A variety of nanomaterials have been developed for the effective
in vivo
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in vivo
delivery of short/ small RNAs, messenger RNAs, and RNAs required for gene editing technologies including clustered regularly interspaced palindromic repeat (CRISPR)/Cas. This review outlines the challenges of delivering RNA therapeutics, explores the chemical synthesis of RNA modifications and carriers, and describes the efforts to design nanomaterials that can be used for a variety of clinical indications.</abstract><cop>Beijing</cop><pub>Tsinghua University Press</pub><doi>10.1007/s12274-018-2099-4</doi><tpages>28</tpages></addata></record> |
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| source | SpringerNature Journals |
| subjects | Atomic/Molecular Structure and Spectra Biomedicine Biotechnology Cellular apoptosis susceptibility protein Chemical modification Chemical synthesis Chemistry and Materials Science Condensed Matter Physics CRISPR Design modifications Gene silencing Genetic modification Genome editing Immunogenicity Intracellular Materials Science Molecular weight Nanomaterials Nanotechnology Organic chemistry Review Article Ribonucleic acid RNA siRNA Transcription |
| title | Design of synthetic materials for intracellular delivery of RNAs: From siRNA-mediated gene silencing to CRISPR/Cas gene editing |
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