• Title/Summary/Keyword: therapeutic potential

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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.

microRNA biomarkers in cystic diseases

  • Woo, Yu Mi;Park, Jong Hoon
    • BMB Reports
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    • v.46 no.7
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    • pp.338-345
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    • 2013
  • microRNAs (miRNAs) are small non-coding RNAs that regulate gene expression by targeting the 3'-untranslated region of multiple target genes. Pathogenesis results from defects in several gene sets; therefore, disease progression could be prevented using miRNAs targeting multiple genes. Moreover, recent studies suggest that miRNAs reflect the stage of the specific disease, such as carcinogenesis. Cystic diseases, including polycystic kidney disease, polycystic liver disease, pancreatic cystic disease, and ovarian cystic disease, have common processes of cyst formation in the specific organ. Specifically, epithelial cells initiate abnormal cell proliferation and apoptosis as a result of alterations to key genes. Cysts are caused by fluid accumulation in the lumen. However, the molecular mechanisms underlying cyst formation and progression remain unclear. This review aims to introduce the key miRNAs related to cyst formation, and we suggest that miRNAs could be useful biomarkers and potential therapeutic targets in several cystic diseases.

Impact of tumour associated macrophages in pancreatic cancer

  • Mielgo, Ainhoa;Schmid, Michael C.
    • BMB Reports
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    • v.46 no.3
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    • pp.131-138
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    • 2013
  • During cancer progression, bone marrow derived myeloid cells, including immature myeloid cells and macrophages, progressively accumulate at the primary tumour site where they contribute to the establishment of a tumour promoting microenvironment. A marked infiltration of macrophages into the stromal compartment and the generation of a desmoplastic stromal reaction is a particular characteristic of pancreatic ductal adenocarcinoma (PDA) and is thought to play a key role in disease progression and its response to therapy. Tumour associated macrophages (TAMs) foster PDA tumour progression by promoting angiogenesis, metastasis, and by suppressing an anti-tumourigenic immune response. Recent work also suggests that TAMs contribute to resistance to chemotherapy and to the emergence of cancer stem-like cells. Here we will review the current understanding of the biology and the pro-tumourigenic functions of TAMs in cancer and specifically in PDA, and highlight potential therapeutic strategies to target TAMs and to improve current therapies for pancreatic cancer.

The Effect of Natural Mate Tea Extract on the Body Weight and Biochemical Biomarker in High Fat Diet-Obese(ob/ob) Mice (천연마테차 물추출물의 고지방식이 비만쥐의 체중과 생화학적 변화에 미치는 영향)

  • Kim, Sang-Tae;Hwang, Cho-Won;Kim, Young-Kyoon
    • YAKHAK HOEJI
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    • v.56 no.1
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    • pp.1-8
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    • 2012
  • In this study, we investigated the anti-obese activity of sevennight extract in high fat diet-ob/ob C57BL/6J mice by oral administered for 1 weeks. Mate water extract (MATEWi) was found to lower whole body and epididymal adipose tissue weights and lowered plasma levels of triglyceride (TG) and total cholesterol (TC), HDL and LDL, compared to those in high fat fed ob/ob group. These results suggest that Mate extract ameliorates obesity through activation of lipogenic enzymes and FA oxidation resulting from phosphorylation of AKT and GSK-$3{\beta}$, and could be developed as a potential therapeutic agent for obese mices.

Design, Syntheses, and Conformational Study of Angiogenesis Inhibitors

  • Park, Kyoungsoo;Dongha Baek;Dongyeol Lim;Park, Sang-Don;Kim, Min-Young;Park, Yong-Sun;Kim, Yangmee
    • Proceedings of the Korean Biophysical Society Conference
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    • 2001.06a
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    • pp.30-30
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    • 2001
  • Since anti-angiogenesis could lead to the suppression of tumor growth, angiogenesis inhibitors have received particular attention for their therapeutic potential. In this study, two angiogenic inhibitors using the bioactive sequence from the kringle 5, AK1(KLYDY), AK2(KLWDF) were designed and synthesized. We have investigated their solution structures using NMR spectroscopy and their activities as angiogenesis inhibitors.(omitted)

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Inhibition of Osteoclast Differentiation by Tanshinones from the Root of Salvia miltiorrhiza Bunge

  • Lee Song-Yi;Choi Doo-Youn;Woo Eun-Rhan
    • Archives of Pharmacal Research
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    • v.28 no.8
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    • pp.909-913
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    • 2005
  • We screened natural products to find compounds with anti-osteoporotic potential using a coculture-based system by which osteoclast differentiation is effectively achieved. We found that methylene chloride soluble fraction of the root of Salvia miltiorrhiza Bunge (Labiatae) suppressed osteoclast differentiation. Five tanshinones, tanshinone IIA (1), tanshinone I (2), cryptotanshinone (3), 15,16-dihydrotanshinone I (4), and ferruginol (5) were subsequently isolated from fraction. Among the five compounds, compounds 1-4 reduced the formation of TRAP­positive multinuclear osteoclasts. These results suggest that the identified tanshinones may be useful candidates for development of therapeutic agents to treat osteoporosis and other bone-resorptive diseases.

CD137-CD137 Ligand Interactions in Inflammation

  • Kwon, Byung-Suk
    • IMMUNE NETWORK
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    • v.9 no.3
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    • pp.84-89
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    • 2009
  • The main stream of CD137 studies has been directed to the function of CD137 in $CD8^+$ T-cell immunity, including its anti-tumor activity, and paradoxically the immunosuppressive activity of CD137, which proves to be of a great therapeutic potential for animal models of a variety of autoimmune and inflammatory diseases. Recent studies, however, add complexes to the biology of CD137. Accumulating is evidence supporting that there exists a bidirectional signal transduction pathway for the CD137 receptor and its ligand (CD137L). CD137/CD137L interactions are involved in the network of hematopoietic and nonhematopoietic cells in addition to the well characterized antigen-presenting cell-T cell interactions. Signaling through CD137L plays a critical role in the differentiation of myeloid cells and their cellular activities, suggesting that CD137L signals trigger and sustain inflammation. The overall consequence might be that the amplified inflammation by CD137L enhances the T-cell activity together with CD137 signals by upregulating costimulatory molecules, MHC molecules, cell adhesion molecules, cytokines, and chemokines. Solving this outstanding issue is urgent and will have an important clinical implication.

Discovery of Novel 11β-HSD1 Inhibitors by Pharmacophore-Based Virtual Screening

  • Kim, Nam-Doo;Lee, Youn-Ho;Han, Chang-Kyun;Ahn, Soon-Kil
    • Bulletin of the Korean Chemical Society
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    • v.33 no.7
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    • pp.2365-2368
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    • 2012
  • The $11{\beta}$-hydroxysteroid dehydrogenase type 1 ($11{\beta}$-HSD1) enzyme is involved in modulation of glucocorticoid activity within target tissues. This enzyme may contribute to obesity and/or metabolic disease through its action in adipose or liver tissue. Inhibition of $11{\beta}$-HSD1 has major therapeutic potential for glucocorticoid-associated diseases, including obesity, diabetes (wound healing), and muscle atrophy. To develop such therapeutics, we performed a pharmacophore-based virtual screening (VS) for identification of novel $11{\beta}$-HSD1 inhibitors and found that the VS hit compounds show potent inhibition of $11{\beta}$-HSD1 enzyme activity. Further, we present a binding model for active compounds. The proposed pharmacophore may serve as a useful guideline for future design of new chemical entities as $11{\beta}$-HSD1-targeted antidiabetic agents.

Diagnostic Evaluation Methods in TMJ Balancing Medicine (턱관절균형의학의 기본 진단검사법)

  • Lee, Young-Jun;Yin, Chang Shik
    • Journal of TMJ Balancing Medicine
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    • v.3 no.1
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    • pp.27-30
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    • 2013
  • Diagnostic evaluation methods frequently adopted in Temporomandibular Balancing Medicine (TBM) are briefly reviewed as to their basic concepts and procedures. TBM diagnostic evaluation methods may be classified into direct and indirect ones. Indirect methods are to observe any change in the manifestations with regard to the (1) posture, (2) pain, (3) muscle strength, and (4) stretching. Direct methods are to evaluate through (1) cervical palpation test, (2) restricted cervical rotation test, and (3) lateral cervical tension test. These methods may provide clinicians valuable information on the current yinyang balance and potential therapeutic factors in relation with the temporomandibular joint, a meridian system, and the whole body.

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Crystal Structure of Human TRAIL, the First Molecule With Selective Antitumor Activity

  • Cha, Sun-Shin;Shin, Hang-Cheol;Oh, Byung-Ha
    • Proceedings of the Korean Biophysical Society Conference
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    • 1999.06a
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    • pp.34-34
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    • 1999
  • TRAIL (also known as Apo-2L) is a newly identified cytokine belonging to the large tumor necrosis factor (TNF) family. TRAIL is a novel molecule in that it induces apoptosis in a wide variety of tumor cells but not in normal cells. In order to help elucidating its biological roles and designing mutants with improved therapeutic potential, we have determined the crystal structure of human TRAIL.(omitted)

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