Development of Targeted Therapies Based on Gene Modification

  • Taylor M. Benson
  • Fatjon Leti
  • Johanna K. DiStefanoEmail author
Part of the Methods in Molecular Biology book series (MIMB, volume 1706)


With the advent of next-generation sequencing (NGS) and the demand for a personalized healthcare system, the fields of precision medicine and gene therapy are advancing in new directions. There is a push to identify genes that contribute to disease development, either alone or in conjunction with other genes or environmental factors, and then design targeted therapies based on this knowledge, rather than the traditional approach of treating generalized symptoms with pharmaceuticals in a one-size-fits-all manner. Identification of genes that contribute to disease pathogenesis and progression is critical for the maturation of the precision medicine field. Concomitant with a better understanding of disease pathology, precision medicine approaches can be adopted with greater confidence and are expected to lead to a new standard for clinical practice. In this chapter, we provide a brief introduction to precision medicine, discuss the importance of identifying genes and genetic variants that contribute to disease development and progression, offer examples of approaches that can be applied to treat specific diseases, and present some of the current challenges and limitations of precision medicine.

Key words

Precision medicine Personalized medicine Gene therapy Pharmacogenomics NGS, GWAS 


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Copyright information

© Springer Science+Business Media, LLC 2018

Authors and Affiliations

  • Taylor M. Benson
    • 1
  • Fatjon Leti
    • 1
  • Johanna K. DiStefano
    • 2
    Email author
  1. 1.Department of Biomedical Research, Center for Genes, Environment, and HealthNational Jewish HealthDenverUSA
  2. 2.Translational Genomics Research InstitutePhoenixUSA

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