Gene Therapy

  • Hao Wu
  • Ram I. Mahato


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.


Human Immunodeficiency Virus Gene Therapy Cystic Fibrosis Transmembrane Conductance Regulator Insertional Mutagenesis Nonviral Vector 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.



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


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Further Reading

  1. Schleef M et al. (2001) Plasmids for therapy cbrsand vaccination. Wiley-VCH, New York, NYGoogle Scholar
  2. Schleef M (2005) DNA pharmaceuticals: formulation and delivery in gene therapy, DNA vaccination and immunotherapy. John Wiley & Sons, HobokenGoogle Scholar
  3. National Institutes of Health (2006) Regenerative medicine. National Institutes of Health, Bethesda, DCGoogle Scholar
  4. Narang A, Mahato RI (2010) Targeted delivery of small and macromolecular drugs. CRC press, Boca Raton, FLGoogle Scholar

Copyright information

© Springer Science+Business Media New York 2013

Authors and Affiliations

  1. 1.Department of Pharmaceutical SciencesUniversity of Tennessee Health Science CenterMemphisUSA
  2. 2.Department of Pharmaceutical Sciences, College of PharmacyUniversity of Nebraska Medical CenterOmahaUSA

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