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Current Status of the Clinical Development of Gene Therapy
Biomed. Sci. Lett. 2018;24:157-167
Published online September 30, 2018;
© 2018 The Korean Society For Biomedical Laboratory Sciences.

Sun-Il Kwon†,*

Department of Biomedical Laboratory Science, Deagu Health Science University, Daegu 41453, Korea
Correspondence to: Sun-Il Kwon. Department of Biomedical Laboratory Science, Deagu Health Science University, Youngsong-ro 15 (Taejeon-dong), Buk-ku, Daegu 41453, Korea.
Tel: +82-53-320-1870, Fax: +82-53-320-1450, e-mail:
Received July 10, 2018; Revised September 3, 2018; Accepted September 4, 2018.
This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License ( which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
The concept of gene therapy is to treat a disease by transferring therapeutic nucleic acids to a patient's cells. It took several decades from the basic theoretical proposal of gene therapy to the current promising treatment option for some important human diseases. The encountered adverse effects in the early clinical studies boosted the development of sophisticated vectors and elaborate clinical designs. The gene therapy is now considered to have the potential to cure many diseases that are incurable with conventional medications. By the end of 2017, about 2,600 clinical trials of gene therapy have been performed or are ongoing for a variety of diseases such as cancers, monogenic diseases, cardiovascular diseases and neurological diseases etc. Here, we present a brief introduction of technical achievement in relation to gene therapy development, and a review of the current status of global gene therapy clinical development.
Keywords : Gene therapy, Cancer, Clinical study, Vector, Target diseases, Guidelines