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Role of Lipid-Based and Polymer-Based Non-Viral Vectors in Nucleic Acid Delivery for Next-Generation Gene Therapy
The field of gene therapy has experienced an insurgence of attention for its widespread ability to regulate gene expression by targeting genomic DNA, messenger RNA, microRNA, and short-interfering RNA for treating malignant and non-malignant disorders. Numerous nucleic acid analogs have been develop...
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| Published in: | Molecules (Basel, Switzerland) Switzerland), 2020-06, Vol.25 (12), p.2866 |
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| Main Authors: | , , , , , |
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| container_issue | 12 |
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| container_title | Molecules (Basel, Switzerland) |
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| creator | Wahane, Aniket Waghmode, Akaash Kapphahn, Alexander Dhuri, Karishma Gupta, Anisha Bahal, Raman |
| description | The field of gene therapy has experienced an insurgence of attention for its widespread ability to regulate gene expression by targeting genomic DNA, messenger RNA, microRNA, and short-interfering RNA for treating malignant and non-malignant disorders. Numerous nucleic acid analogs have been developed to target coding or non-coding sequences of the human genome for gene regulation. However, broader clinical applications of nucleic acid analogs have been limited due to their poor cell or organ-specific delivery. To resolve these issues, non-viral vectors based on nanoparticles, liposomes, and polyplexes have been developed to date. This review is centered on non-viral vectors mainly comprising of cationic lipids and polymers for nucleic acid-based delivery for numerous gene therapy-based applications. |
| doi_str_mv | 10.3390/molecules25122866 |
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Numerous nucleic acid analogs have been developed to target coding or non-coding sequences of the human genome for gene regulation. However, broader clinical applications of nucleic acid analogs have been limited due to their poor cell or organ-specific delivery. To resolve these issues, non-viral vectors based on nanoparticles, liposomes, and polyplexes have been developed to date. This review is centered on non-viral vectors mainly comprising of cationic lipids and polymers for nucleic acid-based delivery for numerous gene therapy-based applications.</description><identifier>ISSN: 1420-3049</identifier><identifier>EISSN: 1420-3049</identifier><identifier>DOI: 10.3390/molecules25122866</identifier><identifier>PMID: 32580326</identifier><language>eng</language><publisher>Switzerland: MDPI AG</publisher><subject>Acids ; Analogs ; Ataxia ; Cationic polymerization ; Deoxyribonucleic acid ; Design of experiments ; DNA ; DNA - genetics ; DNA - therapeutic use ; Efficiency ; Ethanol ; Expression vectors ; Gene expression ; Gene regulation ; Gene therapy ; Gene Transfer Techniques ; Genetic Therapy - trends ; Genetic Vectors - genetics ; Genetic Vectors - therapeutic use ; Genomes ; Humans ; lipid nanoparticles ; Lipids ; Lipids - genetics ; Lipids - therapeutic use ; Liposomes ; miRNA ; mRNA ; Nanoparticles ; Nanoparticles - chemistry ; Nucleic acids ; Physiology ; Polyethylene glycol ; Polymers ; Polymers - chemistry ; Polymers - therapeutic use ; Proteins ; Review ; RNA, Small Interfering - genetics ; RNA, Small Interfering - therapeutic use ; Therapeutic applications ; Vectors (Biology)</subject><ispartof>Molecules (Basel, Switzerland), 2020-06, Vol.25 (12), p.2866</ispartof><rights>2020. 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This review is centered on non-viral vectors mainly comprising of cationic lipids and polymers for nucleic acid-based delivery for numerous gene therapy-based applications.</description><subject>Acids</subject><subject>Analogs</subject><subject>Ataxia</subject><subject>Cationic polymerization</subject><subject>Deoxyribonucleic acid</subject><subject>Design of experiments</subject><subject>DNA</subject><subject>DNA - genetics</subject><subject>DNA - therapeutic use</subject><subject>Efficiency</subject><subject>Ethanol</subject><subject>Expression vectors</subject><subject>Gene expression</subject><subject>Gene regulation</subject><subject>Gene therapy</subject><subject>Gene Transfer Techniques</subject><subject>Genetic Therapy - trends</subject><subject>Genetic Vectors - genetics</subject><subject>Genetic Vectors - therapeutic use</subject><subject>Genomes</subject><subject>Humans</subject><subject>lipid nanoparticles</subject><subject>Lipids</subject><subject>Lipids - genetics</subject><subject>Lipids - therapeutic use</subject><subject>Liposomes</subject><subject>miRNA</subject><subject>mRNA</subject><subject>Nanoparticles</subject><subject>Nanoparticles - chemistry</subject><subject>Nucleic acids</subject><subject>Physiology</subject><subject>Polyethylene glycol</subject><subject>Polymers</subject><subject>Polymers - chemistry</subject><subject>Polymers - therapeutic use</subject><subject>Proteins</subject><subject>Review</subject><subject>RNA, Small Interfering - genetics</subject><subject>RNA, Small Interfering - therapeutic use</subject><subject>Therapeutic applications</subject><subject>Vectors (Biology)</subject><issn>1420-3049</issn><issn>1420-3049</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2020</creationdate><recordtype>article</recordtype><sourceid>COVID</sourceid><sourceid>PIMPY</sourceid><sourceid>DOA</sourceid><recordid>eNplkU1vEzEQhi0EoqXwA7ggS5wX_L3rC1JpoVSKAkKlV8trj1tHm3Vq71bk3-OQULXi5PF45pl5_SL0lpIPnGvycZ0GcPMAhUnKWKfUM3RMBSMNJ0I_fxQfoVelrAhhVFD5Eh1xJjvCmTpGdz8rA6eAF3ETffPZFvDYjh7_SMN2DfmQWaaxuY7ZDvga3JRywXHEy9kNEB0-ddHjcxjiPeQtDinjJfyemgsYIdspphHvQnx1W6-b7Wv0ItihwJvDeYJ-ff1ydfatWXy_uDw7XTROSj01lLaq5y1XivbEqY4r2TOruRJEa1WF2M4RLXVwVLhOWcpIT7jygVvdtkHwE3S55_pkV2aT49rmrUk2mr-JlG-MzVOsEgwhutVKa6Dai04Gq4QCErzsWABoobI-7VmbuV-DdzBO9S-eQJ--jPHW3KR703KpCNst8_4AyOluhjKZVZrzWPUbJmjbMdkSWavovsrlVEqG8DCBErNz3PzneO1593i1h45_FvM_faqoIw</recordid><startdate>20200622</startdate><enddate>20200622</enddate><creator>Wahane, Aniket</creator><creator>Waghmode, Akaash</creator><creator>Kapphahn, Alexander</creator><creator>Dhuri, Karishma</creator><creator>Gupta, Anisha</creator><creator>Bahal, Raman</creator><general>MDPI AG</general><general>MDPI</general><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>3V.</scope><scope>7X7</scope><scope>7XB</scope><scope>88E</scope><scope>8FI</scope><scope>8FJ</scope><scope>8FK</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>AZQEC</scope><scope>BENPR</scope><scope>CCPQU</scope><scope>COVID</scope><scope>DWQXO</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>K9.</scope><scope>M0S</scope><scope>M1P</scope><scope>PIMPY</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>PRINS</scope><scope>5PM</scope><scope>DOA</scope><orcidid>https://orcid.org/0000-0002-0276-9169</orcidid></search><sort><creationdate>20200622</creationdate><title>Role of Lipid-Based and Polymer-Based Non-Viral Vectors in Nucleic Acid Delivery for Next-Generation Gene Therapy</title><author>Wahane, Aniket ; 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| ispartof | Molecules (Basel, Switzerland), 2020-06, Vol.25 (12), p.2866 |
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| source | Open Access: PubMed Central; Publicly Available Content (ProQuest); Coronavirus Research Database |
| subjects | Acids Analogs Ataxia Cationic polymerization Deoxyribonucleic acid Design of experiments DNA DNA - genetics DNA - therapeutic use Efficiency Ethanol Expression vectors Gene expression Gene regulation Gene therapy Gene Transfer Techniques Genetic Therapy - trends Genetic Vectors - genetics Genetic Vectors - therapeutic use Genomes Humans lipid nanoparticles Lipids Lipids - genetics Lipids - therapeutic use Liposomes miRNA mRNA Nanoparticles Nanoparticles - chemistry Nucleic acids Physiology Polyethylene glycol Polymers Polymers - chemistry Polymers - therapeutic use Proteins Review RNA, Small Interfering - genetics RNA, Small Interfering - therapeutic use Therapeutic applications Vectors (Biology) |
| title | Role of Lipid-Based and Polymer-Based Non-Viral Vectors in Nucleic Acid Delivery for Next-Generation Gene Therapy |
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