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Clinical Features, Molecular Analysis, and Outcome of ERT in Korean Patients with Mucopolysaccharidosis Type VI (국내 6형 뮤코다당증의 임상 양상, 분자유전학적 특징 및 효소치료의 효과에 대한 고찰)

  • Wichajarn, Khunton;Kim, Jinsup;Yang, Aram;Sohn, Young Bae;Lee, Beom Hee;Yoo, Han-Wook;Cho, Sung Yoon;Jin, Dong-Kyu
    • Journal of The Korean Society of Inherited Metabolic disease
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    • v.16 no.1
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    • pp.24-33
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    • 2016
  • Mucopolysaccharidosis type VI (MPS VI) is a rare disease caused by the mutation of ARSB with prevalence range from 1/5,000 in northeast Brazil to 1/2,057,529 births in Czech Republic. In Asia, there is only one published figure in Taiwan of about 1/833,000 births. The exact prevalence in the Korean population is unknown, but we estimated the incidence of MPS VI is about 0.03/100,000 live births. Enzyme replacement therapy (ERT) with recombinant human Arylsulfatase B (rhASB) is a modality for the treatment of MPS VI that reduces the excretion of urine glycosaminoglycan (GAG) and improves joint motion, pulmonary function, and endurance. We presented the clinical features, molecular analysis and outcome of ERT in three Korean MPS VI patients. All patients had the typical characteristic clinical features of MPS IV. Short stature, dysostosis multiplex, corneal opacity and valvular heart disease were found at first presentation, while restrictive lung disease and carpal tunnel syndrome developed later in all patients. Molecular analysis demonstrated novel missense and nonsense mutation in the patients, including p.Ile 67Ser, p.Gly328Arg, $p.Arg191^*$, p.Asp352Asn, and p.Gly17Asp. After ERT, urine GAG was decreased in all patients. Skeletal involvement, corneal opacity, heart valve abnormalities and pulmonary function were not improved with ERT, but it had a better outcome on regarding joint motion and endurance. One patient underwent allogeneic bone marrow transplantation (BMT) prior to ERT, but their clinical response was not improved much after BMT. This study demonstrates clinical phenotypes and molecular analysis of the severe form of MPS VI in Korean patients.

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The Changes of Occludin in Tight Junction of Blood-Brain Barrier by ROS (치밀이음부 구조단백질인 Occludin에 대한 활성산소종의 영향)

  • Lee, Hee-Sang;Kim, Dae-Jin;Sohn, Dong-Suep;Jeong, Bong-Su;Choi, Hyung-Taek;Sim, Kyu-Min;Lee, Keum-Jeong;Cho, Hye-Jin;Kim, Suk-Joong;Lee, Jong-Chan;Jeong, Yoon-Hee;Kim, Sung-Su;Lee, Won-Bok
    • Applied Microscopy
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    • v.34 no.4
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    • pp.231-239
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    • 2004
  • Cerebral microvessel endothelial cells that form blood-brain barrier (BBB) have tight junction for maintaining brain homeostasis. Occludin, one of tight junction protein, is crucial for BBB function. $H_2O_2$ induced occludin changes and effects in bovine brain BBB endothelial cells were examined in this study. The decrease of transendothelial electrical resistance (TEER) by $H_2O_2$ was due to disruption of occludin localization. Cytotoxicity test revealed that $H_2O_2$ did not cause cell death below 1 mM $H_2O_2$ within 4 hr. $H_2O_2$ caused intermittent disruption and loss of occludin at tight junctions and occludin disappeared with dose dependent manner from tight junction in confocal laser microscopy. But Western blot revealed that the total amounts of occludin increased by $H_2O_2$ administration. Transmission electron microscopy revealed that the ultrastructure of tight junction was not changed by $H_2O_2$. These data suggest that functional disruption of BBB by $H_2O_2$ was due to the localized loss of occludin in tight junction, but the expression of occludin increased in order to compensate the disrupted function in BBB.

A study on the Hematologic Effects of Radioactive Iodine($^{131}I$) Therapy on Various Thyroid Diseases (방사성동위원소옥소(放射性同位元素沃素)($^{131}I$)의 치료량(治療量)이 갑상선종환자(甲狀腺腫患者)의 혈액상(血液像)에 미치는 영향(影響)에 관(關)한 연구(硏究))

  • Kim, Myung-Jae;Kim, Noe-Kyeong;Lee, Jung-Sang;Choi, Keun-Chul;Lee, Ryong-Woo;Kim, Kee-Won;Kang, Shin-Il
    • The Korean Journal of Nuclear Medicine
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    • v.3 no.1
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    • pp.51-58
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    • 1969
  • To clarify the hematologic effects of the radioiodine ($^{131}I$) in therapeutic doses ($5{\sim}10$ mCi) on the various thyroid patients, authors studied the peripheral blood pictures of 396 goitrous patients before and after radioiodine ($^{131}I$) administrations in the Isotope Clinic of Seoul National University Hospital. Among these 396 cases of goiters, we gave 5 to 10 mCi of radioiodine ($^{131}I$) with single or fractionated administrations. The blood pictures of peripheral blood were repeated after 3 months in 40 cases of 65 cases who had been treated with $^{131}I$. The blood pictures of non-treated thyroid patients were compared with that of normal Korean values to clarify any difference between normal and goiter. The blood pictures of hyperthyroid patients treated with $^{131}I$ therapy were compared with the blood pictures of non-treated thyroid patients. The results were as following: 1) The incidence according to type: Toxic diffuse goiter: 35.4% Nontoxic nodular goiter: 29.7% Euthyroid: 13.8% Nontoxic diffuse goiter: 12.6% Hypothyroidism: 4.3% Thyroiditis($\bar{s}$ subacute form): 1.8% Toxic nodular goiter: 1.4% Malignancy: 1.0% 2) Age incidence: The range of distribution was 11 to 71 years. The peak incidence was found in the 4th decade of life. $80.6{\sim}82.6%$ of those 396 cases were found among the 3rd, 4th and the 5th decades of life. 3) Sex incidence: Sex ratio of male:female was 1:7.8. 4) The most outstanding findings in peripheral blood before treatment were decreased erythrocyte count and hemoglobin value in all types of thyroid diseases, especially in. the cases of hypothyroidism and thyroiditis. Hook worm-infested patients showed no significant difference in erythrocytes and hemoglobin values from those of other hook worm free patients. 5) Total leukocytes count was within normal range. Differential count of W.B.C. showed increased percentile of lymphocyte in diffuse toxic goiter and thyroiditis. 6) 39 cases of diffuse goiter treated with $^{131}I$ toxic showed amelioration in the anemia and restoration to normal range of lymphocyte count in association with increased percentile of neutrophiles 3 months after administration, except a case of toxic nodular goiter. One can observe anemia in slight degree, and increased lymphocytes count in hypothyroidism. Therapeutic dose of radioiodine ($^{131}I$) does not result any residual effect on the hematopoietic function. Radioiodine ($^{131}I$) therapy resulted in improvement of thyroid function in association of amelioration of pevious abnormal blood pictures. 7) Authors did not observe any myxedema resulted from radioiodine therapy during the 3 months period in this study.

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Potential of River Bottom and Bank Erosion for River Restoration after Dam Slit in the Mountain Stream

  • Kang, Ji-Hyun;So, Kazama
    • Proceedings of the Korea Water Resources Association Conference
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    • 2011.05a
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    • pp.46-46
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    • 2011
  • Severe sediment erosion during floods occur disaster and economic losses, but general sediment erosion is basic mechanism to move sediment from upstream to downstream river. In addition, it is important process to change river form. Check dam, which is constructed in mountain stream, play a vital role such as control of sudden debris flow, but it has negative aspects to river ecosystem. Now a day, check dam of open type is an alternative plan to recover river biological diversity and ecosystem through sediment transport while maintaining the function of disaster control. The purpose of this paper is to verify sediment erosion progress of river bottom and bank as first step for river restoration after dam slit by cross-sectional shear stress and critical shear stress. Study area is upstream reach of slit check dam in mountain stream, named Wasada, in Japan. The check dam was slit with two passages in August, 2010. The transects were surveyed for four upstream cross-sections, 7.4 m, 34 m, 86 m, and 150 m distance from dam in October 2010. Sediment size was surveyed at river bottom and bank. Sediment of cobble size was found at the wetted bottom, and small size particles of sand to medium gravel composed river bank. Discharge was $2.5\;m^3/s$ and bottom slope was 0.027 m/m. Excess shear stress (${\tau}_{ex}$) was calculated for hydraulic erosion by subtracting the values of critical shear stress (${\tau}_{c}$) from the value of shear stress (${\tau}$) at river bottom and bank (${\tau}_{ex}=\tau-{\tau}_c$). Shear stress of river bottom (${\tau}_{bottom}$) was calculated using the cross-sectional shear stress, and bank shear stress (${\tau}_{bank}$) was calculated from the method of Flintham and Carling (1988). $${\tau}_{bank}={\tau}^*SF_{bank}((B+P_{bed})/(2^*P_{bank}))$$ where $SF_{bank}=1.77(P_{bed}/p_{bank}+1.5)^{-1.4}$, B is the water surface width, $P_{bed}$ and $P_{bank}$ are wetted parameter of the bed and bank. Estimated values for ${\tau}_{bottom}$ for a flow of $2.5\;m^3/s$ were lower as 25.0 (7.5 m cross-section), 25.7 (34 m), 21.3 (86 m) and 19.8 (150 m), in N/$m^2$, than critical shear stress (${\tau}_c=62.1\;N/m^2$) with cobble of 64 mm. The values were insufficient to erode cobble sediment. In contrast, even if the values of ${\tau}_{bank}$ were lower than the values for ${\tau}_{bottom}$ as 18.7 (7.5 m), 19.3 (34 m), 16.1 (86 m) and 14.7 (150 m), in N/$m^2$, excess shear stresses were calculated at the three cross-sections of 7.5 m, 34 m, and 86 m distances compare with ${\tau}_c$ is 15.5 N/$m^2$ of 16mm gravel. Bank shear stresses were sufficient for erosion of the medium gravel to sand. Therefore there is potential to erode lateral bank than downward erosion in a flow of $2.5\;m^3/s$. Undercutting of the wetted bank can causes bank scour or collapse, therefore this channel has potential to become wider at the same time. This research is about a potential of sediment erosion, and the result could not verify with real data. Therefore it need next step for verification. In addition an erosion mechanism for river restoration is not simple because discharge distribution is variable by snow-melting or rainy season, and a function for disaster control will recover by big precipitation event. Therefore it needs to consider the relationship between continuous discharge change and sediment erosion.

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Biological Functions of N- and O-linked Oligosaccharides of Equine Chorionic Gonadotropin and Lutropin/Chorionic Gonadotropin Receptor

  • Min, K.S.
    • Korean Journal of Animal Reproduction
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    • v.24 no.4
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    • pp.357-364
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    • 2000
  • Members of the glycoprotein family, which includes CG, LH, FSH and TSH, comprise two noncovalently linked $\alpha$- and $\beta$-subunits. Equine chorionic gonadotropin (eCG), known as PMSG, has a number of interesting and unique characteristics since it appears to be a single molecule that possesses both LH- and FSH-like activities in other species than the horse. This dual activity of eCG in heterologous species is of fundamental interest to the study of the structure-function relationships of gonadotropins and their receptors. CG and LH $\beta$ genes are different in primates. In horse, however, a single gene encodes both eCG and eLH $\beta$ -subunits. The subunit mRNA levels seem to be independently regulated and their imbalance may account for differences in the quantities of $\alpha$ - and $\beta$-subunits in the placenta and pituitary. The dual activities of eCG could be separated by removal of the N-linked oligosaccharide on the $\alpha$-subunit Asn 56 or CTP-associated O-linked oligosaccharides. The tethered-eCG was efficiently secreted and showed similar LH-like activity to the dimeric eCG. Interestingly, the FSH-like activity of the tethered-eCG was increased markedly in comparison with the native and wild type eCG. These results also suggest that this molecular can implay particular models of FSH-like activity not LH-like activity in the eCG/indicate that the constructs of tethered molecule will be useful in the study of mutants that affect subunit association and/or secretion. A single-chain analog can also be constructed to include additional hormone-specific bioactive generating potentially efficacious compounds that have only FSH-like activity. The LH/CG receptor (LH/CGR), a membrane glycoprotein that is present on testicular Leydig cells and ovarian theca, granulosa, luteal, and interstitial cells, plays a pivotal role in the regulation of gonadal development and function in males as well as in nonpregnant and pregnant females. The LH/CGR is a member of the family of G protein-coupled receptors and its structure is predicted to of a large extracellular domain connected to a bundle of seven membrane-spanning a-helices. The LH/CGR phosphorylation can be induced with a phorbol ester, but not with a calcium ionophore. The truncated form of LHR also was down-regulated normally in response to hCG stimulation. In contrast, the cell lines expressing LHR-t631 or LHR-628, the two phosphorylation-negative receptor mutant, showed a delay in the early phase of hCG-induced desensitization, a complete loss of PMA-induced desensitization, and an increase in the rate of hCG-induced receptor down-regulation. These results clearly show that residues 632~653 in the C-terminal tail of the LHR are involved in PMA-induced desensitization, hCG-induced desensitization, and hCG-induced down-regulation. Recently, constitutively activating mutations of the receptor have been identified that are associated with familial male-precocious puberty. Cells expressing LHR-D556Y bind hCG with normal affinity, exhibit a 25-fold increase in basal cAMP and respond to hCG with a normal increase in cAMP accumulation. This mutation enhances the internalization of the free and agoinst-occupied receptors ~2- and ~17- fold, respectively. We conclude that the state of activation of the LHR can modulate its basal and/or agonist-stimulated internalization. Since the internalization of hCG is involved in the termination of hCG actions, we suggest that the lack of responsiveness detected in cells expressing LHR-L435R is due to the fast rate of internalization of the bound hCG. This statement is supported by the finding that hCG responsiveness is restored when the cells are lysed and signal transduction is measured in a subcellular fraction (membranes) that cannot internalize the bound hormone.

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Ventilatory Dynamics According to Bronchial Stenosis in Bronchial Anthracofibrosis (기관지 탄분 섬유화증에서 동반된 기관지 협착에 따른 환기역학)

  • Jung, Seung Wook;Kim, Yeon Jae;Kim, Gun Hyun;Kim, Min Seon;Son, Hyuk Soo;Kim, Jun Chul;Ryu, Hyon Uk;Lee, Soo Ok;Jung, Chi Young;Lee, Byung Ki
    • Tuberculosis and Respiratory Diseases
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    • v.59 no.4
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    • pp.368-373
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    • 2005
  • Background : Bronchial anthracofibrosis usually manifest as a form of obstructive airway disease, and can be accompanied by parenchymal diseases such as pneumonia, and pulmonary tuberculosis. This study investigated the ventilatory dynamics according to the severity of bronchial stenosis in patients with bronchial anthracofibrosis. Method : One hundred and thirteen patients with bronchial anthracofibrosis that was confirmed by bronchoscopy and who had undergone a pulmonary function test were enrolled in this study group. The correlation coefficients between the pulmonary functional parameters and the number of lobes with bronchial stenosis were investigated. Results : The incidence of ventilatory dysfunction was 56(49.6%) for obstructive, 8(7.1%) for restrictive, 2(1.8%) for mixed, and 47(41.6%) for a normal pattern. The $FEV_1/FVC$, $FEF_{25{\sim}75%}$, $FEF_{25%}$, $FEF_{50%}$, $FEF_{75%}$, and PEF showed a significant negative correlation (p<0.05) and the Raw had a significant positive correlation with the number of lobes with bronchial stenosis(p<0.001). Conclusion : These findings suggest that the most common abnormality of the ventilatory function in bronchial anthracofibrosis is an obstructive pattern with a small airway dysfunction according to the severity of bronchial stenosis.

A Study on the Village Improvement Plan by Typological Analysis of Greenbelt-lifted Villages (개발제한구역 해제취락 유형분석을 통한 취락정비방안 연구)

  • Yoon, Jeong-Joong;Choi, Sang-Hee
    • Land and Housing Review
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    • v.4 no.1
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    • pp.77-87
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    • 2013
  • About 1,800 villages have released from Greenbelt since Greenbelt-reform-policy for readjustment of the area was promoted after 1997. Even though the government intended to attract planned development & improvement of these lifted villages through District Unit Plan and designating the lifted area as low-rise and low-density zoning considering the characteristics of the Greenbelt region, there are still many problems to be solved: a lack of funds, insufficient capability for self-improvement and unexecuted SOCs in long-term etc. It seems that these problems are caused by focusing on the lifting areas itself instead of researching deeply the condition and characteristics of the villages and searching proper direction/plans of improvement before lifting Greenbelt In addition, the existing plan of village improvement and management was not considering physical and spacial characteristics of the areas, social and economic situation of residents and relationship between the villages and surrounding cities, though these conditions are different among each villages, and the related regulations are applied uniformly across all the villages and those have been causing many civil appeals and environmental problems. In these respects, this study aims to consider the problems of the lifted villages using the existing researches on them and to make typology by characteristics-data of the villages and to establish improvement strategies of each types. In this study, the villages were classified into 5 types as a result of cluster analysis on 424 villages among all 1,800 through variables of locational potentiality : location, accessibility, size and form of village, condition of regulations etc. According to function of the villages, they were divided into 4 types: urban-type, rural-type, industrial-type and neighborhood-centered-type. This study also drew 4 improvement-strategy-types by combination of locational potentiality and village-function : type of improving life-environment, type of improving production-infra, type of inducing-planned-improvement and type of constructing center-of living-circle. Finally, this study suggested the directions of the each 4 types to desirable improvement and management which could be used to make and complement plans for village improvement.

Technical Inefficiency in Korea's Manufacturing Industries (한국(韓國) 제조업(製造業)의 기술적(技術的) 효율성(效率性) : 산업별(産業別) 기술적(技術的) 효율성(效率性)의 추정(推定))

  • Yoo, Seong-min;Lee, In-chan
    • KDI Journal of Economic Policy
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    • v.12 no.2
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    • pp.51-79
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    • 1990
  • Research on technical efficiency, an important dimension of market performance, had received little attention until recently by most industrial organization empiricists, the reason being that traditional microeconomic theory simply assumed away any form of inefficiency in production. Recently, however, an increasing number of research efforts have been conducted to answer questions such as: To what extent do technical ineffciencies exist in the production activities of firms and plants? What are the factors accounting for the level of inefficiency found and those explaining the interindustry difference in technical inefficiency? Are there any significant international differences in the levels of technical efficiency and, if so, how can we reconcile these results with the observed pattern of international trade, etc? As the first in a series of studies on the technical efficiency of Korea's manufacturing industries, this paper attempts to answer some of these questions. Since the estimation of technical efficiency requires the use of plant-level data for each of the five-digit KSIC industries available from the Census of Manufactures, one may consture the findings of this paper as empirical evidence of technical efficiency in Korea's manufacturing industries at the most disaggregated level. We start by clarifying the relationship among the various concepts of efficiency-allocative effciency, factor-price efficiency, technical efficiency, Leibenstein's X-efficiency, and scale efficiency. It then becomes clear that unless certain ceteris paribus assumptions are satisfied, our estimates of technical inefficiency are in fact related to factor price inefficiency as well. The empirical model employed is, what is called, a stochastic frontier production function which divides the stochastic term into two different components-one with a symmetric distribution for pure white noise and the other for technical inefficiency with an asymmetric distribution. A translog production function is assumed for the functional relationship between inputs and output, and was estimated by the corrected ordinary least squares method. The second and third sample moments of the regression residuals are then used to yield estimates of four different types of measures for technical (in) efficiency. The entire range of manufacturing industries can be divided into two groups, depending on whether or not the distribution of estimated regression residuals allows a successful estimation of technical efficiency. The regression equation employing value added as the dependent variable gives a greater number of "successful" industries than the one using gross output. The correlation among estimates of the different measures of efficiency appears to be high, while the estimates of efficiency based on different regression equations seem almost uncorrelated. Thus, in the subsequent analysis of the determinants of interindustry variations in technical efficiency, the choice of the regression equation in the previous stage will affect the outcome significantly.

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Public Park Awareness along with Community Garden Cultivation Participation within an Urban Park (도시공원 내 텃밭 경작 참여 여부에 따른 공원 공공성 인식 연구)

  • Nam Koong, Hee;Kim, Seul-Yea;Kang, Eun-Jee;Kim, Yong-Geun
    • Journal of the Korean Institute of Landscape Architecture
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    • v.43 no.1
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    • pp.120-131
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    • 2015
  • This research aimed at understanding recognition of the park's community garden, public evaluation of it, and user awareness of the public enhancement plan. This was based on the problem that arose in the park community garden as public awareness research along with community garden cultivation participation within the urban park. In order to compare recognition differences along with community garden cultivation participation, the researcher conducted an analysis by dividing research participants into a cultivation participant group and non-participant group. As a result first, the cultivation participant group positively recognized the necessity of the community garden compared to the non-participant group. However, they recognized the community garden as a space for private profit, which threatened the public aspect of the park. Second, as a result of evaluating community garden publicness, the non-participant group which was alienated from community garden use, had a negative opinion about the community garden. Third, as a result of researching awareness of the community garden distribution method and consumption to promote publicness of the community garden, the cultivation participant group recognized the community garden as a space for private profit. Therefore, it is necessary for both groups to be aware of the public value of the community garden among the park users for sustainable management and operation. The significance of this research is the direction in the aspect of users to form, manage and operate the community garden in the urban park without damaging publicness of the park or conflicting between the function of the park and the function of the community garden. Henceforth, the researcher expects that this research can be utilized to maintain publicness of the park in respect of formation of the community garden in the park and management and operation.

THE ROLE OF TYPE 2 DIABETES AS A PREDISPOSING RISK FACTOR ON THE PULPO-PERIAPICAL PATHOGENESIS: REVIEW ARTICLE (치수 치근단 병소의 전구 위험요인으로서의 제 2 형 당뇨의 역할에 관한 소고)

  • Kim, Jin-Hee;Bae, Kwang-Shik;Seo, Deog-Gyu;Hong, Sung-Tae;Lee, Yoon;Hong, Sam-Pyo;Kum, Kee-Yeon
    • Restorative Dentistry and Endodontics
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    • v.34 no.3
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    • pp.169-176
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    • 2009
  • Diabetes Mellitus (DM) is a syndrome accompanied with the abnormal secretion or function of insulin, a hormone that plays a vital role in controlling the blood glucose level (BGL). Type land 2 DM are most common form and the prevalence of the latter is recently increasing, The aim of this article was to assess whet her Type 2 DM could act as a predisposing risk factor on the pulpo-periapical pathogenesis. Previous literature on the pathologic changes of blood vessels in DM was thoroughly reviewed. Furthermore, a histopathologic analysis of artificially-induced periapical specimens obtained from Type 2 diabetic and DM-resistant rats was compared. Histopathologic results demonstrate that the size of periapical bone destruction w as larger and the degree of pulpal inflammation was more severe in diabetic rats, indicating that Type 2 D M itself can be a predisposing risk factor that makes the host more susceptible to pulpal infection. The possible reasons may be that in diabetic state the lumen of pulpal blood vessels are thickened by atheromatous deposits, and microcirculation is hindered, The function of polymorphonuclear leukocyte is also impair ed and the migration of immune cells is blocked, leading to increased chance of pulpal infection. Also, lack of collateral circulation of pulpal blood vessels makes the pulp more susceptible to infection. These decrease the regeneration capacity of pulpal cells or tissues, delaying the healing process, Therefore, when restorative treatment is needed in Type 2 DM patients, dentists should minimize irritation to the pulpal tissue un der control of BGL.