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Gene Therapy: A Pharmacokinetic/Pharmacodynamic Modelling Overview

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ABSTRACT

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.

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Authors and Affiliations

  1. Department of Pharmacy and Pharmaceutical Technology School of Pharmacy, University of Navarra, Pamplona, Spain

    Zinnia P. Parra-Guillén & Iñaki F. Trocóniz

  2. Division of Hepatology and Gene Therapy, Center for Investigation in Applied Medicine (CIMA), Pamplona, Spain

    Gloria González-Aseguinolaza & Pedro Berraondo

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  1. Zinnia P. Parra-Guillén
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  2. Gloria González-Aseguinolaza
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  3. Pedro Berraondo
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  4. Iñaki F. Trocóniz
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Correspondence to Iñaki F. Trocóniz.

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Parra-Guillén, Z.P., González-Aseguinolaza, G., Berraondo, P. et al. Gene Therapy: A Pharmacokinetic/Pharmacodynamic Modelling Overview. Pharm Res 27, 1487–1497 (2010). https://doi.org/10.1007/s11095-010-0136-4

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  • DOI: https://doi.org/10.1007/s11095-010-0136-4

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