Journal of Pharmaceutical Investigation

The Korean Society of Pharmaceutical Sciences and Technology (한국약제학회)
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Current Status of Gene Therapy as a New Drug Delivery System

신약전달기술체계인 유전자 치료의 현재까지의 개발동향

  • Bae, Yun-Sung (College of Engineering, Institute of Biotechnology, Department of Bioscience and Biotechnology, Sejong University,Microbiology Laboratory, Quality Control Department, Kyung Dong Pharm. Co. Ltd.) ;
  • Cho, Jung-Yoon (College of Engineering, Institute of Biotechnology, Department of Bioscience and Biotechnology, Sejong University) ;
  • Ji, Sang-Mi (College of Engineering, Institute of Biotechnology, Department of Bioscience and Biotechnology, Sejong University) ;
  • Lee, Young-Joo (College of Engineering, Institute of Biotechnology, Department of Bioscience and Biotechnology, Sejong University)
  • 배윤성 (세종대학교 생명공학과,경동제약) ;
  • 조정윤 (세종대학교 생명공학과) ;
  • 지상미 (세종대학교 생명공학과) ;
  • 이영주 (세종대학교 생명공학과)
  • Published : 2002.09.20
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Abstract

Gene therapy is fundamentally a sophisticated drug delivery technology to cure a disease by the transfer of genetic material to modify living cells. In other words, the gene is used as a therapeutic drug much like a chemical compound is employed in chemotherapy. Currently almost 600 clinical trials are underway worldwide since the first clinical trials carried out in 1990 to treat adenosine deaminase deficiency using retroviral vectors. Despite the great progress still is there no gene therapy product being approved as a new drug. This is partly due to a lack of an ideal gene delivery system that is safe and can provide stable, optimal level production of the therapeutic proteins in the cell. This review covers the current status of several different biological and physico-chemical agents that are being developed as gene delivery vehicles. Although gene therapy promises great hopes toward the cure of a broad spectrum of genetic and acquired diseases, the success of gene therapy heavily asks for the development of vector systems for safe and efficient application in humans.

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