• Title, Summary, Keyword: gene therapy

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Current trends of stem cell-mediated gene therapy (줄기 세포 분야의 유전자 치료 연구 동향)

  • Oh, Yu-Kyoung;Chung, Hyung-Min
    • Journal of Pharmaceutical Investigation
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    • v.32 no.2
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    • pp.65-72
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    • 2002
  • Recently, stem cell-mediated gene therapy is emerging as a novel therapeutic approach. For the successful gene modification of stem cells, the development of a suitable gene transfer technique needs to be preceded. This review focuses on the various gene transfer techniques based on nonviral and viral vectors, and physical methods. The advantages and disadvantages of each gene transfer method are compared, and the general properties of these vectors are discussed in relation to the gene transfer in stem cell research. This review also highlights the therapeutic application of stem cell-mediated gene therapy. The choice of gene transfer vectors may vary depending on the type of the stem cells and the target of stem cell therapy. Of various gene transfer methods, viral vector-based gene therapy has been emphasized due to the higher transfection efficiency. The current status and up-to-date findings of stem cell-mediated gene therapy are discussed in the viewpoint of the various targets of stem cell therapy such as the modification of stem cell potency, the acceleration of regeneration process and the formation of expressional organization.

Tumor targeted gene therapy (종양 표적 유전자 치료)

  • Kang, Joo-Hyun
    • Nuclear Medicine and Molecular Imaging
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    • v.40 no.5
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    • pp.237-242
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    • 2006
  • Knowledge of molecular mechanisms governing malignant transformation brings new opportunities for therapeutic intervention against cancer using novel approaches. One of them is gene therapy based on the transfer of genetic material to an organism with the aim of correcting a disease. The application of gene therapy to the cancer treatment has led to the development of new experimental approaches such as suicidal gene therapy, inhibition of oncogenes and restoration of tumor-suppressor genes. Suicidal gene therapy is based on the expression in tumor cells of a gene encoding an enzyme that converts a prodrug into a toxic product. Representative suicidal genes are Herpes simplex virus type 1 thymidine kinase (HSV1-tk) and cytosine deaminase (CD). Especially, physicians and scientists of nuclear medicine field take an interest In suicidal gene therapy because they can monitor the location and magnitude, and duration of expression of HSV1-tk and CD by PET scanner.

Gene-Editing: Interpretation of Current Law and Legal Policy

  • Kim, Na-Kyoung
    • Development and Reproduction
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    • v.21 no.3
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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.

Monitoring Gene Therapy by Radionuclide Approaches (핵의학적 기법을 이용한 유전자 치료 영상법)

  • Min, Jung-Joon
    • Nuclear Medicine and Molecular Imaging
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    • v.40 no.2
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    • pp.96-105
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    • 2006
  • Molecular imaging has its root in nuclear medicine and gene therapy monitoring. Therefore, recent progress in the development of non-invasive imaging technologies, particularly nuclear medicine, should allow molecular imaging to play a major role in the field of gene therapy. These tools have recently been validated in gene therapy models for continuous quantitative monitoring of the location, magnitude, and time-variation of gene delivery and/or expression. This article reviews the use of radionuclide imaging technologies as they have been used in imaging gene delivery and gene expression for gene therapy applications. The studios published to date lend support that noninvasive imaging tools will help to accelerate pre-clinical model validation as well as allow for clinical monitoring of human gene therapy.

Current Status of the Clinical Development of Gene Therapy

  • Kwon, Sun-Il
    • Biomedical Science Letters
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    • v.24 no.3
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    • pp.157-167
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    • 2018
  • 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.