• 제목/요약/키워드: Gene therapy

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Gene Medicine : A New Field of Molecular Medicine

  • Kim, Chong-Kook;Haider, Kh-H;Lim, Soo-Jeong
    • Archives of Pharmacal Research
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    • 제24권1호
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    • pp.1-15
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    • 2001
  • Gene therapy has emerged as a new concept of therapeutic strategies to treat diseases which do not respond to the conventional therapies. The principle of gene therapy is to Introduce genetic materials into patient cells to produce therapeutic proteins in these cells. Gene therapy is now at the stage where a number of clinical trials have been carried out to patients with gene-deficiency disease or cancer. Genetic materials for gene therapy are generally composed of gene expression system and gene delivery system. For the clinical application of gene therapy in a way which conventional drugs are used, researches have been focused on the design of gene delivery system which can offer high transfection efficiency with minimal toxicity. Currently, viral delivery systems generally provide higher transfection efficiency compared with non-viral delivery systems while non-viral delivery systems are less toxic, less immunogenic and manufacturable in large scale compared with viral systems. Recently, novel strategies towards the design of new non-viral delivery system, combination of viral and non-viral delivery systems and targeted delivery system have been extensively studied. The continued effort in this area will lead us to develop gene medicine as "gene as a drug" in the near future.

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Gene Therapy for Oral Cancer

  • Chung, In-Jae
    • Biomolecules & Therapeutics
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    • 제15권4호
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    • pp.273-280
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    • 2007
  • New treatment approaches are needed to improve the effectiveness of oral cancer treatment, since surgical resection of the tumor in oral region causes various oral dysfunctions. The molecular biology of oral cancer has been progressively delineated. Concurrently, gene therapy techniques have been developed that allow targeting or replacement of dysfunctional genes in cancer cells, offering the potential to treat a wide range of cancer. Oral carcinoma is attractive target for gene therapy because of its accessibility. In this article, we review the current status of gene therapy as applied to oral carcinoma.

Gene-Editing: Interpretation of Current Law and Legal Policy

  • Kim, Na-Kyoung
    • 한국발생생물학회지:발생과생식
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    • 제21권3호
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    • pp.343-349
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    • 2017
  • tWith the development of the third-generation gene scissors, CRISPR-Cas9, concerns are being raised about ethical and social repercussions of the new gene-editing technology. In this situation, this article explores the legislation and interpretation of the positive laws in South Korea. The BioAct does not specify and regulate 'gene editing' itself. However, assuming that genetic editing is used in the process of research and treatment, we can look to the specific details of the regulations for research on humans as well as gene therapy research in order to see how genetic editing is regulated under the BioAct. BioAct differentiates the regulation between (born) humans and embryos etc. and the regulation differ entirely in the manner and scope. Moreover, due to the fact that gene therapy products are regarded as drugs, they fall under different regulations. The Korean Pharmacopoeia Act put stringent sanctions on clinical trials for gene therapy products and the official Notification "Approval and Examination Regulations for Biological Products, etc." by Food and Drug Safety Administration may be applied to gene editing for gene therapy purposes.

Liposome-Mediated Cancer Gene Therapy: Clinical Trials and their Lessons to Stem Cell Therapy

  • Lee, Jung-Hoon;Lee, Min-Jae
    • Bulletin of the Korean Chemical Society
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    • 제33권2호
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    • pp.433-442
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    • 2012
  • The promise of stem cell therapy for various clinical applications seems getting realistic. An increasing number of researchers, from virtually every discipline of natural sciences, are flocking into this new world. Only ten years ago, gene therapy was the medicine for the 21st century. The possibility was endless. Although the science itself underlying gene therapy was very young, the field was exploding under the optimism that this new medicine would revolutionize both the basic and clinical sciences. For many reasons, the initial target was cancer. Here, we will focus on the results of cancer gene therapy clinical trials using liposome or nonviral gene carrier, hoping that the lesson from here will be a guideline for the new generation of cell-based therapies.

Adenovirus vs AAV Vectors for Gene Delivery: Their Advantages and Disadvantages

  • Im Dong-Soo
    • 한국미생물학회:학술대회논문집
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    • 한국미생물학회 2002년도 추계학술대회
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    • pp.109-115
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    • 2002
  • Gene therapy is to treat and cure diseases by an introduction of therapeutic genes in defective cells or tissues of human body. Gene delivery system, gene expression system, and therapeutic gene are three core elements for gene therapy. The efficient delivery of therapeutic genes and appropriate gene expression are the crucial issues for therapeutic outcome of gene delivery. Because it can be used in common for the treatment and cure of various diseases, gene delivery system is the most important core element for a successful gene therapy. Viruses are naturally evolved to transfer their genomes into host cells efficiently. This ability has made vectorologists exploit viruses as attractive vehicles for the delivery of therapeutic genes. Viral vectors based on adenovirus (Ad) and adeno-associated virus (AAV) have been often used for gene delivery in laboratory. Ad and AAV vectors derived from human DNA viruses differ greatly in their life cycle, expression level and duration of transgenes, immunogenicity, and vector preparation. Both vectors can be used as effective tools for gene therapy and more recently in functional genomics. Here, the characteristics of Ad and AAV vectors are discussed.

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