• Title/Summary/Keyword: gene therapy

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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.

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.

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.