• Title/Summary/Keyword: Gene therapy

검색결과 907건 처리시간 0.026초

암 유전자 치료제의 개발 현황 (Cancer Gene Therapy. History and Major Developments)

  • 정인재
    • Toxicological Research
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    • 제19권3호
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    • pp.247-257
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    • 2003
  • Medicine is undergoing a revolution in the understanding of the mechanisms through which disease processes develop. The advent of genetics and molecular biology to oncology not only is providing surrogate predictors of therapy response and survival which are forming the basis for selection among established treatment options, but is providing targets for new directions in therapy as well. Molecular modification of somatic cells for the purposes of protecting the normal cells from the toxicity of cancer chemotherapy, for the sensitization of the tumor cells to therapy and use of conditionally replicating viral vector have been new directions of cancer treatment which have reached the clinical arena. Advances in molecular pharmacology and vector design summarized in this paper may provide solutions to some of the existing problems in the technology of gene transfer therapy. Continued basic research into the biological basis of human disease, systemic studies of the application of these discoveries to therapy and the improvement of vector for gene delivery all combined may result in advances in this important field of therapy over the next few years.

Nervonic Acid Inhibits Replicative Senescence of Human Wharton's Jelly-Derived Mesenchymal Stem Cells

  • Sun Jeong Kim;Soojin Kwon;Soobeen Chung;Eun Joo Lee;Sang Eon Park;Suk-Joo Choi;Soo-Young Oh;Gyu Ha Ryu;Hong Bae Jeon;Jong Wook Chang
    • International Journal of Stem Cells
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    • 제17권1호
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    • pp.80-90
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    • 2024
  • Cellular senescence causes cell cycle arrest and promotes permanent cessation of proliferation. Since the senescence of mesenchymal stem cells (MSCs) reduces proliferation and multipotency and increases immunogenicity, aged MSCs are not suitable for cell therapy. Therefore, it is important to inhibit cellular senescence in MSCs. It has recently been reported that metabolites can control aging diseases. Therefore, we aimed to identify novel metabolites that regulate the replicative senescence in MSCs. Using a fecal metabolites library, we identified nervonic acid (NA) as a candidate metabolite for replicative senescence regulation. In replicative senescent MSCs, NA reduced senescence-associated 𝛽-galactosidase positive cells, the expression of senescence-related genes, as well as increased stemness and adipogenesis. Moreover, in non-senescent MSCs, NA treatment delayed senescence caused by sequential subculture and promoted proliferation. We confirmed, for the first time, that NA delayed and inhibited cellular senescence. Considering optimal concentration, duration, and timing of drug treatment, NA is a novel potential metabolite that can be used in the development of technologies that regulate cellular senescence.

국내 유전자치료 의약품 개발 및 평가 시 고려해야 할 사항 (Points To Consider in Development and Evaluation of the Gene Therapy Products in Korea)

  • 김진석
    • Journal of Pharmaceutical Investigation
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    • 제30권2호
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    • pp.139-143
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    • 2000
  • Gene therapy is becoming a very promising and feasible medical intervention as the understanding of human diseases extends to their molecular levels. Since the first US Food and Drug Administration (FDA)-approved human gene therapy protocol was approved in 1990, over 300 human clinical trial protocols had been approved worldwide so far. Even though some of the domestic gene therapy clinical trials also proved promising and more are awaiting, it should be emphasized that many safety aspects as well as effectiveness aspects should be considered during the development process. Moreover, there seems to be less restricted guidelines from the National Control Authority (NCA) in initiating human clinical trials. This article is intended to suggest some basis and points to consider in the development and evaluation of gene therapy products including antisense oligonucleotides pharmaceuticals.

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Targeted Polymeric Gene Delivery for Anti-angiogenic Tumor Therapy

  • Kim, Won-Jong;Kim, Sung-Wan
    • Macromolecular Research
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    • 제15권2호
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    • pp.100-108
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    • 2007
  • Gene therapy has become a promising strategy for the treatment of genetically based diseases, such as cancer, which are currently considered incurable. A major obstacle in the field of cancer gene therapy is the development of a safe and efficient delivery system for therapeutic gene transfer. Non-viral vectors have attracted great interest, as they are simple to prepare, stable, easy to modify and relatively safe compared to viral vectors. In this review, an insight into the strategies developed for polyethylenimine (PEI)-based non-viral vectors has been provide, including improvement of the polyplex properties by incorporating hydrophilic spacer, poly(ethylene glycol) (PEG). Moreover, this review will summarize the strategies for the tumor targeting. Specifically, a targeted polymeric gene delivery system, PEI-g-PEG-RGD, will be introduced as an efficient gene delivery vector for tumor therapy, including its functional analysis both in vitro and in vivo.

폐암의 유전자 치료법을 위한 암특이적인 PRC1 프로모터 (A Cancer-specific Promoter for Gene Therapy of Lung Cancer, Protein Regulator of Cytokinesis 1 (PRC1))

  • 조영화;윤혜진;권희충;김희종;조성하;강봉수;김연주;설원기;박기랑
    • 생명과학회지
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    • 제18권10호
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    • pp.1395-1399
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    • 2008
  • 우리는 최근에 PRC1 프로모터가 유방암 유전자치료를 위하여 전사 표적이 된 유전자의 발현을 조절할 수 있는 후보 프로모터임을 보고하였다. 우리는 본 실험에서 PRC1이 폐암유전자 치료에서도 적용이 가능한지 조사하였다. 특정 프로모터가 루시퍼라제 유전자와 연결된 플라스미드를 이용한 형질전환 assay에서 PRC1 프로모터는 정상폐세포주에서는 활성을 보이지 않으나 폐암세포주에선 약 30 배의 활성을 보였다. 이는 이미 암특이적인 발현으로 알려진 BIRC5 (survivin) 프로모터와 유사한 결과였다. 또한, 바이러스 벡터를 이용한 실험에서 PRC1은 CMV 프로모터에 비해 아데노부속바이러스에서 약 75%, 아데노바이러스에서 약 66%의활성을 보였다. 이와 대조적으로, PRC1 프로모터를 함유한 이 들 두 종류의 바이러스는 정상 폐세포에서는 20%정도의 낮은 활성을 보였다. 흥미롭게도, 인간 폐종양세포를 이식한 생쥐모델을 사용한 결과에서는 PRC1 프로모터가 CMV 프로모터와 비슷한 생체 활성을 보였다. 종합하면, 이상의 결과는 PRC1이 폐암 유전자치료를 위한 전사표적 유전자의 발현을 위한 프로모터로 사용 가능함을 암시한다.

척수 운동신경원의 기능과 관련된 생존운동신경원 단백질의 역할 (The Role of Survival Motor Neuron Protein associated with Function of Spinal Motor Neuron)

  • 송주영;권영실;남기원;송주민;김동현;김석범;문동철;최진호;김진상
    • The Journal of Korean Physical Therapy
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    • 제13권2호
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    • pp.433-444
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    • 2001
  • This review highlights the ontogenesis and the differentiation of motor neuron in spinal cord, and introduce the survival motor neuron(SMN) which is associated with growth and survival of motor neurons. The differentiation of floor plate cells and motor neurons in the vertebrate neural tube appears to be induced by signals from the notochord. This signal is Sonic hedgehog(Shh). The early development of motor neurons involves the inductive action of Shh. The SMN gene is essential for embryonic viability. SMN mRNA is also expressed in virtually all cell types in spinal cord, including large motor neurons. The SMN protein is involved in RNA processing and during early embryonic development is necessary fer cell survival. Two SMN genes are present in 5q 13 in humans: the telomeric gene(SMNt), which is the SMA-determining gene, and the centromeric analog gene(SMNc). The majority of transcripts from the SMNt gene are full length but, major transcripts of the SMNc gene have a high degrees of alternative splicing and tend to have little or no exon 7. The SMN is involved in the RNA processing(the biogenesis of snRNPs and pre-mRNA splicing), the anti-apoptotic effects, and regulating gene expression.

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