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  • Print publication year: 2019
  • Online publication date: October 2019

Chapter 52 - Gene Therapy: Principles and Clinical Potential

from Gene Therapy

Summary

Gene therapy uses a vector to deliver a gene to its required site, where expression of the protein can produce a therapeutic effect. In the last decade there have been significant therapeutic breakthroughs, with clinical trials of postnatal gene therapy showing efficacy for a variety of diseases, such as hemophilia, congenital blindness, congenital immunodeficiency and neuromuscular disorders, and the first gene therapy for familial hyperlipidemia was approved in the European Union (EU) in 2012.