Gene Therapy: A Pharmacokinetic/Pharmacodynamic Modelling Overview

被引：32

作者：

Parra-Guillen, Zinnia P. ^{[1
]}

Gonzalez-Aseguinolaza, Gloria ^{[2
]}

Berraondo, Pedro ^{[2
]}

Troconiz, Inaki F. ^{[1
]}

机构：

[1] Univ Navarra, Sch Pharm, Dept Pharm & Pharmaceut Technol, E-31080 Pamplona, Spain

[2] CIMA, Div Hepatol & Gene Therapy, Pamplona, Spain

来源：

PHARMACEUTICAL RESEARCH | 2010年 / 27卷 / 08期

关键词：

computational modelling; gene therapy; pharmacokinetic/pharmacodynamic modelling; CATIONIC LIPOSOME COMPLEXES; NAKED PLASMID DNA; INTRACELLULAR TRAFFICKING; IN-VIVO; INTRAVENOUS-INJECTION; TRANSGENE EXPRESSION; DELIVERY-SYSTEM; NONLINEAR PHARMACODYNAMICS; TRANSFECTION EFFICIENCY; QUANTITATIVE-ANALYSIS;

D O I：

10.1007/s11095-010-0136-4

中图分类号：

O6 [化学];

学科分类号：

0703 ;

摘要：

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.

引用

页码：1487 / 1497

页数：11

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