Gene Therapy: A Pharmacokinetic/Pharmacodynamic Modelling Overview
Since gene therapy started over 20 years ago, more than one-thousand clinical trials have been carried out. Nonviral vectors present interesting properties for their clinical application, but their efficiency in vivo is relatively low, and further improvements in these vectors are needed. Elucidatin...
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| Veröffentlicht in: | Pharmaceutical research 2010-08, Vol.27 (8), p.1487-1497 |
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| creator | Parra-Guillén, Zinnia P González-Aseguinolaza, Gloria Berraondo, Pedro Trocóniz, Iñaki F |
| description | Since gene therapy started over 20 years ago, more than one-thousand clinical trials have been carried out. Nonviral vectors present interesting properties for their clinical application, but their efficiency in vivo is relatively low, and further improvements in these vectors are needed. Elucidating how nonviral vectors behave at the intracellular level is enlightening for vector improvement and optimization. Model-based approach is a powerful tool to understand and describe the different processes that gene transfer systems should overcome inside the body. Model-based approach allows for proposing and predicting the effect of parameter changes on the overall gene therapy response, as well as the known application of the pharmacokinetic/pharmacodynamic modelling in conventional therapies. The objective of this paper is to critically review the works in which the time-course of naked or formulated DNA have been quantitatively studied or modelled. |
| doi_str_mv | 10.1007/s11095-010-0136-4 |
| format | Article |
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Nonviral vectors present interesting properties for their clinical application, but their efficiency in vivo is relatively low, and further improvements in these vectors are needed. Elucidating how nonviral vectors behave at the intracellular level is enlightening for vector improvement and optimization. Model-based approach is a powerful tool to understand and describe the different processes that gene transfer systems should overcome inside the body. Model-based approach allows for proposing and predicting the effect of parameter changes on the overall gene therapy response, as well as the known application of the pharmacokinetic/pharmacodynamic modelling in conventional therapies. The objective of this paper is to critically review the works in which the time-course of naked or formulated DNA have been quantitatively studied or modelled.</description><identifier>ISSN: 0724-8741</identifier><identifier>EISSN: 1573-904X</identifier><identifier>DOI: 10.1007/s11095-010-0136-4</identifier><identifier>PMID: 20387096</identifier><identifier>CODEN: PHREEB</identifier><language>eng</language><publisher>Boston: Boston : Springer US</publisher><subject>Biochemistry ; Biological and medical sciences ; Biomedical and Life Sciences ; Biomedical Engineering and Bioengineering ; Biomedicine ; Cellular biology ; computational modelling ; Computer based modeling ; Gene therapy ; General pharmacology ; Genetic Therapy - adverse effects ; Genetic Therapy - trends ; Genetic Vectors - adverse effects ; Genetic Vectors - pharmacokinetics ; Humans ; Medical Law ; Medical sciences ; Models, Biological ; Pharmaceutical sciences ; Pharmaceutical technology. Pharmaceutical industry ; pharmacokinetic/pharmacodynamic modelling ; Pharmacology. Drug treatments ; Pharmacology/Toxicology ; Pharmacy ; Review</subject><ispartof>Pharmaceutical research, 2010-08, Vol.27 (8), p.1487-1497</ispartof><rights>Springer Science+Business Media, LLC 2010</rights><rights>2015 INIST-CNRS</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c456t-aac2861bacec57e1f094e73b48c6754196c04805c84732a91b80b079606a477f3</citedby><cites>FETCH-LOGICAL-c456t-aac2861bacec57e1f094e73b48c6754196c04805c84732a91b80b079606a477f3</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/s11095-010-0136-4$$EPDF$$P50$$Gspringer$$H</linktopdf><linktohtml>$$Uhttps://link.springer.com/10.1007/s11095-010-0136-4$$EHTML$$P50$$Gspringer$$H</linktohtml><link.rule.ids>314,780,784,27924,27925,41488,42557,51319</link.rule.ids><backlink>$$Uhttp://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=23207782$$DView record in Pascal Francis$$Hfree_for_read</backlink><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/20387096$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Parra-Guillén, Zinnia P</creatorcontrib><creatorcontrib>González-Aseguinolaza, Gloria</creatorcontrib><creatorcontrib>Berraondo, Pedro</creatorcontrib><creatorcontrib>Trocóniz, Iñaki F</creatorcontrib><title>Gene Therapy: A Pharmacokinetic/Pharmacodynamic Modelling Overview</title><title>Pharmaceutical research</title><addtitle>Pharm Res</addtitle><addtitle>Pharm Res</addtitle><description>Since gene therapy started over 20 years ago, more than one-thousand clinical trials have been carried out. Nonviral vectors present interesting properties for their clinical application, but their efficiency in vivo is relatively low, and further improvements in these vectors are needed. Elucidating how nonviral vectors behave at the intracellular level is enlightening for vector improvement and optimization. Model-based approach is a powerful tool to understand and describe the different processes that gene transfer systems should overcome inside the body. Model-based approach allows for proposing and predicting the effect of parameter changes on the overall gene therapy response, as well as the known application of the pharmacokinetic/pharmacodynamic modelling in conventional therapies. The objective of this paper is to critically review the works in which the time-course of naked or formulated DNA have been quantitatively studied or modelled.</description><subject>Biochemistry</subject><subject>Biological and medical sciences</subject><subject>Biomedical and Life Sciences</subject><subject>Biomedical Engineering and Bioengineering</subject><subject>Biomedicine</subject><subject>Cellular biology</subject><subject>computational modelling</subject><subject>Computer based modeling</subject><subject>Gene therapy</subject><subject>General pharmacology</subject><subject>Genetic Therapy - adverse effects</subject><subject>Genetic Therapy - trends</subject><subject>Genetic Vectors - adverse effects</subject><subject>Genetic Vectors - pharmacokinetics</subject><subject>Humans</subject><subject>Medical Law</subject><subject>Medical sciences</subject><subject>Models, Biological</subject><subject>Pharmaceutical sciences</subject><subject>Pharmaceutical technology. Pharmaceutical industry</subject><subject>pharmacokinetic/pharmacodynamic modelling</subject><subject>Pharmacology. 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| subjects | Biochemistry Biological and medical sciences Biomedical and Life Sciences Biomedical Engineering and Bioengineering Biomedicine Cellular biology computational modelling Computer based modeling Gene therapy General pharmacology Genetic Therapy - adverse effects Genetic Therapy - trends Genetic Vectors - adverse effects Genetic Vectors - pharmacokinetics Humans Medical Law Medical sciences Models, Biological Pharmaceutical sciences Pharmaceutical technology. Pharmaceutical industry pharmacokinetic/pharmacodynamic modelling Pharmacology. Drug treatments Pharmacology/Toxicology Pharmacy Review |
| title | Gene Therapy: A Pharmacokinetic/Pharmacodynamic Modelling Overview |
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