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Gene Therapy

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Pharmaceutical Biotechnology

Abstract

The human body is composed of a variety of proteins. Almost all human diseases are the results of improper production or functioning of proteins. Traditional small molecule drugs usually interact with proteins such as enzymes, hormones, transcriptional factors and even RNA molecules to exert their therapeutic potential. However, many severe and debilitating hereditary diseases (e.g., type I diabetes, hemophilia, cystic fibrosis) and several chronic diseases (e.g., hypertension, ischemic heart disease, asthma, Parkinson’s disease, motor neuron disease, multiple sclerosis) remain inadequately treated by the conventional pharmaceutical approaches. This chapter describes progress made over the years in repairing, turning-off or replacing dysfunctional genes with exogenous DNA as a novel approach to treat, cure, or ultimately prevent disease by changing the expression of a person’s genes.

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Acknowledgements

We would like to thank the National Institutes of Health (NIH) for the financial support (R01DK69968 and R01GM113166).

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Authors and Affiliations

  1. WuXi Biologics, a WuXi AppTec Affiliate, Shanghai, China

    Hao Wu

  2. Department of Pharmaceutical Sciences, College of Pharmacy, University of Nebraska Medical Center, Omaha, NE, USA

    Amit Kumar Chaudhary & Ram I. Mahato

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  1. Hao Wu
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  2. Amit Kumar Chaudhary
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  3. Ram I. Mahato
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Correspondence to Ram I. Mahato .

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Editors and Affiliations

  1. Utrecht University, Utrecht, The Netherlands

    Daan J. A. Crommelin

  2. Faculty of Pharmaceutical Sciences and The Centre for Health Evaluation & Outcomes Sciences (CHEOS), The University of British Columbia (UBC), Vancouver, BC, Canada

    Robert D. Sindelar

  3. College of Pharmacy, University of Tennessee, Health Science Center, Memphis, TN, USA

    Bernd Meibohm

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Wu, H., Chaudhary, A.K., Mahato, R.I. (2019). Gene Therapy. In: Crommelin, D., Sindelar, R., Meibohm, B. (eds) Pharmaceutical Biotechnology. Springer, Cham. https://doi.org/10.1007/978-3-030-00710-2_16

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