Gene Therapy

  • Hao Wu
  • Amit Kumar Chaudhary
  • Ram I. MahatoEmail author


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.


Gene therapy Viral and non-viral vectors Plasmid DNA Minicircle Genome engineering CRISPR-Cas9 



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


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Suggested Reading

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Copyright information

© Springer Nature Switzerland AG 2019

Authors and Affiliations

  1. 1.WuXi Biologics, a WuXi AppTec AffiliateShanghaiChina
  2. 2.Department of Pharmaceutical Sciences, College of PharmacyUniversity of Nebraska Medical CenterOmahaUSA

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