Fabry disease (FD), a rare X-linked lysosomal storage disorder, is caused by mutations in the α-galactosidase A gene gene encoding α-galactosidase A (α-Gal A). The functional deficiency of α-Gal A results in progressive accumulation of neutral glycosphingolipids, causing multi-organ damages including cardiac, renal, cerebrovascular systems. The current treatment is comprised of enzyme replacement therapy (ERT), oral pharmacological chaperone therapy and adjunctive supportive therapy. ERT has been introduced 20 years ago, changing the outcome of FD patients with proven effectiveness. However, FD patients have many unmet needs. ERT needs a life-long intravenous therapy, inefficient bio-distribution, and generation of anti-drug antibodies. Migalastat, a pharmacological chaperone, augmenting α-Gal A enzyme activity only in patients with mutations amenable to the therapy, is now available for clinical practice. Furthermore, these therapies should be initiated before the organ damage becomes irreversible. Development of novel drugs aim at improving the clinical effectiveness and convenience of therapy. Clinical trial of next generation ERT is underway. Polyethylene glycolylated enzyme has a longer half-life and potentially reduced antigenicity, compared with standard preparations with longer dosing interval. Moss-derived enzyme has a higher affinity for mannose receptors, and seems to have more efficient access to podocytes of kidney which is relatively resistant to reach by conventional ERT. Substrate reduction therapy is currently under clinical trial. Gene therapy has now been started in several clinical trials using in vivo and ex vivo technologies. Early results are emerging. Other strategic approaches at preclinical research level are stem cell-based therapy with genome editing and systemic mRNA therapy.
Divyanshoo Rai Kohli;Bashar A. Aqel;Nicole L. Segaran;M. Edwyn Harrison;Norio Fukami;Douglas O. Faigel;Adyr Moss;Amit Mathur;Winston Hewitt;Nitin Katariya;Rahul Pannala
Annals of Hepato-Biliary-Pancreatic Surgery
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v.27
no.1
/
pp.49-55
/
2023
Backgrounds/Aims: Data regarding outcomes of endoscopic retrograde cholangiography (ERC) in liver transplant (LT) recipients with biliary-enteric (BE) anastomosis are limited. We report outcomes of ERC and percutaneous transhepatic biliary drainage (PTBD) as first-line therapies in LT recipients with BE anastomosis. Methods: All LT recipients with Roux-BE anastomosis from 2001 to 2020 were divided into ERC and PTBD subgroups. Technical success was defined as the ability to cannulate the bile duct. Clinical success was defined as the ability to perform cholangiography and therapeutic interventions. Results: A total of 36 LT recipients (25 males, age 53.5 ± 13 years) with Roux-BE anastomosis who underwent biliary intervention were identified. The most common indications for a BE anastomosis were primary sclerosing cholangitis (n = 14) and duct size mismatch (n = 10). Among the 29 patients who initially underwent ERC, technical success and clinical success were achieved in 24 (82.8%) and 22 (75.9%) patients, respectively. The initial endoscope used for the ERC was a single balloon enteroscope in 16 patients, a double balloon enteroscope in 7 patients, a pediatric colonoscope in 5 patients, and a conventional reusable duodenoscope in 1 patient. Among the 7 patients who underwent PTBD as the initial therapy, six (85.7%) achieved technical and clinical success (p = 0.57). Conclusions: In LT patients with Roux-BE anastomosis requiring biliary intervention, ERC with a balloon-assisted enteroscope is safe with a success rate comparable to PTBD. Both ERC and PTBD can be considered as first-line therapies for LT recipients with a BE anastomosis.
Lee, Hee Su;Choi, Jong Myung;Kim, Dae Young;Kim, Seung Yu
Horticultural Science & Technology
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v.34
no.2
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pp.220-227
/
2016
The objective of this research was to investigate the influence of application rates of dolomitic lime in the acid substrate on the mitigation of high bicarbonate damage in 'Seolhyang' strawberry. For this purpose, an acid substrate was formulated by mixing of sphagnum peat moss and pine bark with the ratio of 5:5 (v/v). The pH, EC and CEC of the substrate analyzed before application of dolomite were 4.07, $0.46dS{\cdot}m^{-1}$, and $91.3cmol+/kg^{-1}$, respectively. To adjust the pH of substrate, various amounts of dolomitic lime [$CaMg(CO_3)_2$] such as 0 (control), 1, 2, 3, and $4g{\cdot}L^{-1}$ were added during substrate formulation. Then, seedlings with 3 leaf stage were transplanted as mother plants and those were fed with Hoagland solution containing $240mg{\cdot}L^{-1}$ of the $HCO_3{^-}$. The growth parameters of mother plants 140 days after transplanting, such as plant height, chlorophyll content, and fresh weight were the highest in the treatments of 2 and $3g{\cdot}L^{-1}$ of dolomitic lime. The physiological disorders in mother plants were not observed in the 1, 2 and $3g{\cdot}L^{-1}$ treatments, but the symptoms of Ca, K and B deficiencies were observed in the 0 and $4g{\cdot}L^{-1}$ treatments. During the propagation period, the number of daughter plants derived from each mother plant were 21.0, 29.5, 35.8, 27.3 and 16.0 in the treatments of 0, 1, 2, 3 and $4g{\cdot}L^{-1}$, respectively. The substrate pH during cultivation of mother plants were maintained at appropriate levels for the 1 and $2g{\cdot}L^{-1}$ treatments, whereas it was the highest in $4g{\cdot}L^{-1}$ treatment. The contents of macro- and micro-elements in the above ground tissue were the highest in $2g{\cdot}L^{-1}$ and the lowest in $4g{\cdot}L^{-1}$ lime treatments. Above results suggest that the bicarbonate injury originated from ground water can be mitigated by adjusting the amount of dolomitic lime incorporated into the acid substrate.
This research was conducted to investigate the influence of post-planting fertilizer concentrations on the growth of seedlings and changes of nutrient concentrations of media in tomato seedling production through sub-irrigation. Two root media such as peat moss (grade of 0 to 6 mm, PM06) plus perlite (grade of 1 to 2 mm (PE2)(7:3, v/v) and peat moss (grade of 5 to 15 mm, PM515) plus PE2 (7:3, v/v) were formulated and filled into 72-cell plug trays. After seeds of 'Dotaerang Dia' tomato were sown and germinated at $28^{\circ}C$, the trays were moved to greenhouse and seedlings were raised 35 days. When the cotyledons were emerged, post-planting fertilizers of 13-2-13, 15-0-15 and 20-9-20 ($N-P_2O_5-K_2O$) were applied in a sequence. The fertilizer concentrations based on N in each plug stage were differed with $25mg{\cdot}L^{-1}$ in three treatments. The fertilizer solutions were supplied when the weight of plug trays decreased to 40 to 50% compared to container capacity. The root media were collected in 1, 2, 4, and 5 weeks after sowing and were divided into top, middle, and bottom parts, then were analysed for pH, EC and macro-nutrient concentrations. The seedling growth was investigated 5 weeks after sowing. The pH and EC in PM06+PE2 was higher than those of PM515+PE2. The bottom and mid-part had higher pH and lower EC compared to upper part in each medium. The differences of EC between upper and bottom parts were around 2 times in each medium. The $NH_4-N$ and K concentrations in program 3 of PM06+PE2 showed the highest concentrations among all treatments. The $NO_3-N$ concentrations in PM06+PE2 increased gradually and this rising tendency become severe as post-planting fertilizer concentrations were elevated. The seedling growth in terms of fresh and dry weights was the highest in the treatment of program 2 in PM06+PE2 among all treatments tested. Above results indicate that the gradual increases of fertilizer concentrations from 25 to $125mg{\cdot}L^{-1}$ in plug stages 2, 3, and 4 plug stages are desirable for
Kim, Yong-Gyun;Lee, Byung-Joo;Lee, Sun-Gye;Lee, Byung-Eui
Journal of Mushroom
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v.16
no.3
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pp.147-154
/
2018
This study was aimed to improve the productivity and income of mushroom farming by developing a new casing material as a substitute for clay loam casing soil, which is becoming more difficult to acquire. When the new casing materials were used for the stable production of button mushroom (Agaricus bisporus), a 1:1 mixture of clay loam and button mushroom media obtained after harvest supported 13% greater mycelial growth ($32.0kg/3.3m^2$). This material was better than clay loam soil in preventing contamination with environmental compounds and pests. The use of an inexpensive 1:1 mixture of peat moss and coco peat resulted superior mycelial growth with 4% better yield ($32.9kg/3.3m^2$) compared with conventional clay loam soil. Advantages of these casing materials included ready availability and improved productivity. Mixtures of peat moss + coco peat + zeolite (50%:30%:20%) and coco peat + coal ash (75%:25%) could substitute for conventional casing soil. Additionally, the novel mixtures containing material obtained after cultivation might be used to produce organic fertilizer.
Lee, Ka Youn;Min, Ji Yun;Kim, Mi Sun;Moon, Byeong Cheol;Kang, Young Min
Journal of agriculture & life science
/
v.50
no.2
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pp.33-43
/
2016
Pinellia tripartita(Blume) Schott is a herbaceous perennial plant belonging to the Araceae and distributed on Asia including of Korea, Japan, and China. P. tripartita is often used for gardening but has not been developed mass-breeding methods. In this study, we compared the tuber growth in different combinations of mixed soils used six compositions. Tubers used to study was cultured in vitro and divided into two groups. Type I was diameter more than 1cm and the group of Type II was diameter below than 1cm. Enlargement of tubers and growth of aerial parts were measuring the plant height, number of fresh leaves and dead leaves, number of bullets, tuber size, and fresh / dry weight. The size/weight and numbers of tubers from the mixed soil B (coir 68.0%, peat moss 14.7%, perlite 3.0%, vermiculite 7.0% and zeolite 7.0%) were the best grown up for eight weeks. In case of Type I, GI (Growth index) of tuber size and weight were 45% and 101%, respectively. The difference of growth was doubled compared to the bad growth treatment as the mixed soil E(Coir 14.3%, peat moss 14.3%, perlite 42.9%, vermiculite 14.3%, and zeolite 14.3%). These results could be used as the basic information for the similar experimental design for the P. ternata.
Potted, germinating Pinus rigida ${\times}$ P. taeda seedlings were inoculated with Pisolithus tinctorius (Pt) ectomycorrhizal fungus to test the effectiveness of Pt in relation to organic amendment and changes in soil fertility and soil texture. Pt was cultured as mycelia in vermiculite-peat moss mixture with nutrients and added to sterilized pot soils with or without organic amendment (fully fermented compost) at three soil texture levels (sand, loamy sand, and sandy loam) in a factorial design. Plants were grown in a greenhouse for 4 months and harvested to compare their growth with non-mycorrhizal plants and plants infected by natural fungi. Regardless of sod texture, soil fertility, or organic amendment, seedlings inoculated with Pt were better in dry weight and height than non-mycorrhizal plants or those infected by natural fungi. An exception was observed in the most fertile soil (0.075% N and 1.32% organic matter content in sandy loam with organic amendment), where non-mycorrhizal plants were slightly bigger (8%) and heavier (18%) than Pt-inoculated plants. In over-all average, Pt-inoculated seedlings were 30% taller and 107% heavier than those infected by natural fungi and 31 % taller and 60% heavier than non-mycorrhizal plants. Growth stimulation of seedlings by Pt was more pronounced in less fertile sand soil when organic was not amended. Mycorrhizal frequency of Pt (% of mycorrhizal root tips) was reduced to about half (from 84 to 33% in sandy loam and from 77 to 40% in loamy sand) by organic amendment, while that of natural fungi was not significantly affected. Severe nitrogen deficiency was observed in the needles of non-mycorrhizal plants (1.38% N), while both Pt-inoculated plants (1.68% N) and those infected by natural fungi (1.89% N) did not develop symptom, suggesting an active role of mycorrhizae in absorption of soil nitrogen. Top to root ratio increased with organic amendment to non-mycorrhizal plants, but was not significantly affected by fungal treatment. It was concluded from this study that relative effectiveness of Pt was determined by soil fertility. Organic amendment to less fertile sand soil increased effectiveness of Pt, while the same amendment to more fertile loamy sand and sandy loam decreased effectiveness of Pt. Benefits of Pt mycorrhizae would be expected most either when organic was not added to the soil, or when soil nutrients were not abundant.
Yang, Seung Koo;Son, Dong-Mo;Choi, Kyung Ju;Kim, Sang Chaul;Kim, Wol-Soo;Chung, Soon Ju
Horticultural Science & Technology
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v.19
no.4
/
pp.483-487
/
2001
This study was carried out to investigate the role of attached leaflet on rooting and growth by one nodal cutting in tomato. As cutting sources 4 to 6 nodes could be obtained from one nursery seedling. Medium for cutting was a mixture with perlite plus peat moss (1 : 1, v/v), and each plug capacity was 30mL in the 72 cells-plug tray. Plant height and the number of leaves were significantly increased by attached leaflet cutting in 'Pepe' and 'Coco' cherry tomato. Dry weights of top and root were increased as much as 3 to 15 times in the cutting attached leaflet. Rooting percentage was 93.5% in one node cutting and 86% in the cutting with hypocotyl node part. At 20 to 24 days after nodal cutting, healthy nursery plant could be produced to transplant in field. The nursery plants by nodal cutting (NPNC) showed more fibrous roots and less tap roots than that of seedling. In terms of rooting pattern, NPNC rooted at the whole ground stem part, while rooting of seedling occurred at basal part of hypocotyl.
Kim, Han-Kyoung;Lee, Kang-Hyo;Cheong, Jong-Chun;Jhune, Chang-Sung;Seok, Sun-Ja;Jang, Kab-Yeul
Journal of Mushroom
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v.7
no.1
/
pp.9-21
/
2009
This study was executed in an attempt to investigate a artificial requisites of fruitbody occurrence. Environmental requirements on habitat for fruitbody occurrence of collected cultures resulted in leading to $13-16^{\circ}C$ and 75% relative humidity, and requiring silt loam of soil texture which had more nutritional substances than a dry field. Optimal temperature was $25^{\circ}C$, medium PDB, and pH 5.0 in cultural conditions. Mannose required of 5% in ASI 59002, 59003, 59004, but 3% in ASI 59001 was selected as optimum carbon source. The substrates stimulating sclerotium formation were cotton waste, or cotton waste + oak sawdust (mixture ratio of 8:2), which had 20% additive of wheat barn respectively. Sclerotium was formed well in the substrate adjusted chemical properties by applying 2% of calcium sulfate. Sclerotium formation was the most effective in the treatment of peat moss + oak sawdust (mixture ratio of 5:5) + 30% of wheat barn.
White clover (Trifolium repens L) gives rise to either weak persistence or overdominance in the pastures. To get information on grazing method to surmount the problem, the experiment was done to measure the effect of infrequent, frequent and their alternations on harvest yield and morphological characteristics of the clover. Individual plants of Regal, Louisiana S-l, Grasslands Huia, and Aberystwyth S184 were grown in 22cm plastic pot containing a 2: 1: 1 soil: sand: Peat moss mixture for 27 days after transplanting 50-day seedlings raised on 3cm pots, and then their all fully expanded leaves are trimmed. Defoliation treatments were forced every 1 (CC, frequent), 4 week(RR,infrequent) or their alternations(CR, RC) after 8 weeks from the trimming. To analyze the treatment effects, plants were sampled on 4, 8, 12, and 16 weeks after the trimming. Harvest yield of infrequent defoliation (RR) was higher than that of frequent defoliation (CC). Leaf area and no. of leaves per plant, petiole length and stolon length per plant, moreover, showed the similar result to the yield but stolon length and leaf area per g were reverse. The alternation of infre-quent and then frequent defoliation (RC) had greater yield than that of their reverse (CR) although both alternations showed intermediate ones compared to CC and RR. Morphological characteristics, furthermore, related to the clover leaf were immediately changed by alternations of defoliation inter-val(RC and CR) while those done to the stolon were lasted longer thereafter(RC and CR). Harvest yields in RC and CR were positively correlated to leaf area and no. of leaves or stolon length per plant but negatively done to leaf area and stolon length per g. It is concluded that weak persistence or overdominance of white clover in pasture can be controlled by alternation of infrequent and then frequent defoliation or reverse.
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