Copyright ©The Author(s) 2015. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Med Genet. Feb 27, 2015; 5(1): 1-13
Published online Feb 27, 2015. doi: 10.5496/wjmg.v5.i1.1
Cell mediated gene therapy: A guide for doctors in the clinic
Moon Jong Noh, Ogden Copeland, Michael O’Mara, Kwan Hee Lee
Moon Jong Noh, Ogden Copeland, Michael O’Mara, Kwan Hee Lee, TissueGene Inc., Rockville, MD 20850, United States
Author contributions: Noh MJ wrote most of this paper and gave input on the molecular aspect of TG-C; Copeland O reviewed and wrote the Food and Drug Administration related aspects; O’Mara M wrote the manufacturing part; Lee KH selected the review papers, decided the contents of the paper.
Conflict-of-interest: All of the authors are employees of TissueGene Inc. TissueGene Inc. will pay the fees for publication and editing of this paper. TissueGene Inc. owns the patent rights to protect the commercialization of TG-C.
Open-Access: This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See:
Correspondence to: Kwan Hee Lee, MD, PhD, Chief Medical Officer, TissueGene Inc., 9605 Medical Center Drive, #200, Rockville, MD 20850, United States.
Telephone: +1-301-9216000101 Fax: +1-301-9216011
Received: September 25, 2014
Peer-review started: September 28, 2014
First decision: November 27, 2014
Revised: January 6, 2015
Accepted: January 15, 2015
Article in press: January 19, 2015
Published online: February 27, 2015

The recent approval of gene therapy products in Europe and Asia and the upsurge of gene therapy products in clinical trials signal the rebound of this technology not only for many orphan diseases but also for non-life threatening diseases. Following the success of induced pluripotent stem (iPS) cells in research, other modified ex vivo gene therapies are also knocking on the door of the clinic. Historically, gene therapy has experienced many ups and downs and still faces many challenges. During the past 10 years, many new ideas have been tried, and the goal of making this technology a more effective treatment modality through greater safety and control is coming within reach. The first clinical trial of iPS cells has begun, and cell mediated gene therapy products have reached phase III in some countries. The potential for tumorigenicity and immunogenicity are still concerns with these products, so physicians should understand the biological aspects of engineered cells in the clinic. In this review article, we attempted to provide a summary update of the current state of knowledge regarding this technology: that is, we reviewed products that have finished clinical trials, are still in clinical trials and/or are at the research stage. We also focused on the challenges, future directions, and strategies for making this technology available in the clinic. In addition, the available measures for making gene therapy products safer are within the scope of this article. It is also important to understand the manufacturing process for gene therapy products, because cell characteristics can change during the cell expansion process. When physicians use gene therapy products in the clinic, they should be aware of the viability, temperature sensitivity and stability of these cells because biologic products are different from chemical products. Although we may not be able to answer all possible questions and concerns, we believe that this is the right time for physicians to increase their interest in and understanding of this evolving technology.

Keywords: Cell mediated gene therapy, Review, Physicians, Clinical and research stage, In the clinic

Core tip: In this review article, the authors attempted to provide an up to date summary of the current knowledge regarding cell mediated gene therapy that is, we reviewed products that have finished clinical trial, are in clinical trial and at the research stage. The authors also tried to cover the challenges, future directions, and strategies to make this technology available in the clinic. This is the right time for the physicians to have knowledge of this evolving technology that already reached the bedside.