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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 function of proteins. Traditional small molecule drugs usually interact with proteins such as enzymes, hormones, and transcriptional factors to exert their therapeutic potential. However, many severe and deliberating diseases (e.g., 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.

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Acknowledgements

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

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Authors and Affiliations

  1. Department of Pharmaceutical Sciences, University of Tennessee Health Science Center, 19 S. Manassas, RM 224, Memphis, TN, 38103-3308, USA

    Hao Wu Ph.D.

  2. Department of Pharmaceutical Sciences, College of Pharmacy, University of Nebraska Medical Center, 986025 Nebraska Medical Center, Omaha, NE, 68198-6025, USA

    Ram I. Mahato Ph.D.

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  1. Hao Wu Ph.D.
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Correspondence to Ram I. Mahato Ph.D. .

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  1. Department of Pharmaceutical Sciences, Utrecht University, Utrecht, Utrecht, The Netherlands

    Daan J. A. Crommelin

  2. Department of Pharmaceutical Sciences and Department of Medicine, The University of British Columbia, Vancouver, British Columbia, Canada

    Robert D. Sindelar

  3. Department of Pharmaceutical Sciences, University of Tennessee Health Science Center, College of Pharmacy, Memphis, Tennessee, USA

    Bernd Meibohm

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Wu, H., Mahato, R.I. (2013). Gene Therapy. In: Crommelin, D., Sindelar, R., Meibohm, B. (eds) Pharmaceutical Biotechnology. Springer, New York, NY. https://doi.org/10.1007/978-1-4614-6486-0_24

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