Abstract

Based on the theoretical and clinical development of modern medicines, gene therapy has been a promising treatment strategy for cancer and other diseases. The practice of gene therapy is nearly 27 years old, since the first authorized gene transfer study took place at the National Institute of Health in 1989. However, gene therapy was not readily adopted worldwide, until recently. Several gene therapy clinical trials have been carried out in China since 1998, and medical research in China has flourished. In this report, we review the history of gene therapy in China, focusing on treatment protocol, the administration cycle, dosage calculation, and the evaluation of therapeutic effects, in order to provide more information for the additional development of this promising treatment strategy.

Highlights

  • State Key Laboratory of Oral Diseases, National Clinical Research Center for Oral Diseases, Department of Head and Neck Oncology, West China Hospital of Stomatology, Sichuan University, Academic Editor: Michael Barry

  • Since deoxyribonucleic acid (DNA) was identified as hereditary material, gene transfer has become a focus of medical researchers

  • An adenosine deaminase-severe combined immune deficiencies (ADA-SCID) trial was conducted in the EU by Bordignon C. et al, and those results indicated successful gene transfer in long-lasting progenitor cells, the immune repertoire of the patients was normalized, and their humoral immunities were restored after two years of treatment [6]

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Summary

Brief History of Gene Therapy in the World

Since deoxyribonucleic acid (DNA) was identified as hereditary material, gene transfer has become a focus of medical researchers. An adenosine deaminase-severe combined immune deficiencies (ADA-SCID) trial was conducted in the EU by Bordignon C. et al, and those results indicated successful gene transfer in long-lasting progenitor cells, the immune repertoire of the patients was normalized, and their humoral immunities were restored after two years of treatment [6]. The first p53 recombinant adenovirus was reported by Zhang et al, who introduced WT p53 by Ad5CMV-p53 into four human NSCLC cell lines, in order to show the promising tumor-inhibiting effects of p53 gene therapy [8] Based on these preclinical studies, the first clinical trial of p53 gene therapy was carried out by Roth et al, who directly administrated a retroviral vector containing the WT p53 gene into nine NSCLC patients for whom conventional treatments had failed, and the results showed tumor regression in three of the patients and a stabilization of tumor growth in a further three patients [9].

Distribution
Brief History of Gene Therapy in China
The Combined Therapy and the Sequence of the Treatment Protocol
Dosage Calculation and Routes of Administration in Gene Therapy
Treatment Cycle of the Administration
Evaluation of the Gene Therapy
The Future of Gene Therapy
Conclusions
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