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Chapter 52 - Gene Therapy: Principles and Clinical Potential

from Gene Therapy

Published online by Cambridge University Press:  21 October 2019

By
Suzanne M. K. Buckley  and
Anna L. David
Edited by
Mark D. Kilby ,
Anthony Johnson  and
Dick Oepkes
Mark D. Kilby
Affiliation:
University of Birmingham
Anthony Johnson
Affiliation:
University of Texas Medical School at Houston
Dick Oepkes
Affiliation:
Leids Universitair Medisch Centrum
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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.

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Type
Chapter
Information
Fetal Therapy
Scientific Basis and Critical Appraisal of Clinical Benefits
 , pp. 540 - 560
Publisher: Cambridge University Press
Print publication year: 2020

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