Gene therapy

Hao Wu, Ram I Mahato

Research output: Chapter in Book/Report/Conference proceedingChapter

1 Scopus citations


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.

Original languageEnglish (US)
Title of host publicationPharmaceutical Biotechnology
Subtitle of host publicationFundamentals and Applications, Fourth Edition
PublisherSpringer New York
Number of pages31
ISBN (Electronic)9781461464860
ISBN (Print)9781461464853
Publication statusPublished - Jan 1 2013


ASJC Scopus subject areas

  • Medicine(all)

Cite this

Wu, H., & Mahato, R. I. (2013). Gene therapy. In Pharmaceutical Biotechnology: Fundamentals and Applications, Fourth Edition (pp. 477-507). Springer New York.