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Pharmacodynamic Evaluation: Gene Therapy

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Drug Discovery and Evaluation: Methods in Clinical Pharmacology
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Abstract

Gene therapy is based on the genome modifications by introducing nucleic acids in the cells of the patient to treat diseases. There are different gene therapy strategies, the most common of which is to add a healthy copy of the disease-inducing gene. Other strategies consist of replacing the gene which is responsible for the disease for a healthy copy, or to inactivate a gene that is functioning improperly. Gene therapy is designed for untreatable diseases or with a heavy treatment.

To modify the genome of patients, different gene vectors are used. There are two major classes of them, synthetic vectors which are complexes between nucleic acids (DNA, RNA, or protein) and synthetic molecules and viral vectors which are derived from viruses. Each of them has their advantages and limits.

The vectors can be administered directly in vivo or used for ex vivo approaches which consist to genetically modify the patient cells in vitro before being reintroduced into the patient.

This chapter gives a statement of the art of gene therapy. It describes the different strategies and tools used in this area before describing the seminal clinical trials and the range of diseases for which gene therapy could be relevant. This chapter concludes by considering the issues and challenges of the gene therapy.

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Grandchamp, N. (2019). Pharmacodynamic Evaluation: Gene Therapy. In: Hock, F., Gralinski, M. (eds) Drug Discovery and Evaluation: Methods in Clinical Pharmacology. Springer, Cham. https://doi.org/10.1007/978-3-319-56637-5_51-1

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