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1رسالة جامعية
المؤلفون: Humble, Stewart Wynn
المساهمون: Wade-Martins, Richard, Ryan, Brent, Ward, Michael E.
مصطلحات موضوعية: Neurobiology, Genetics, CRISPR (Genetics), Lysosomes, Molecular neurobiology, Lysosomal storage diseases, Genetic engineering
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2رسالة جامعية
المؤلفون: Bogucka, Agnieszka
المساهمون: Gloster, Tracey
مصطلحات موضوعية: Sulfatases, Heparan sulfate, Lysosomal storage, Fragment based screening, Heparin, Lysosomal storage diseases, Drugs--Design
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3رسالة جامعية
المؤلفون: Prause, Kevin
مرشدي الرسالة: Arenz, Christoph, Fiedler, Dorothea, Berg, Thorsten
مصطلحات موضوعية: Saure Sphingomyelinase, Glucocerebrosidase, Pharmakologische Chaperone, photoaktivierbare Inhibitoren, myo-Inosit, Aminoinosit-Derivate, Lysosomale Speichererkrankungen, Morbus Niemann-Pick, Morbus Gaucher, Zeitaufgelöste Enzyminhibition, Sphingomyelin, Glucosylceramid, acid sphingomyelinase, glucocerebrosidase, pharmacological chaperones, photocaged inhibitors, myo-Inositol, Aminoinositol derivatives, lysosomal storage diseases, time resolved enzyme inhibition, Glucosylceramide, 547 Organische Chemie, ddc:547
وصف الملف: application/pdf
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4رسالة جامعية
المؤلفون: Sáenz Díaz, Laura Camila
المساهمون: Guevara Morales, Johana María, Echeverri Pena, Olga Yaneth
مصطلحات موضوعية: Lisosoma, fibroblastos, leucocitos, monocitos, macrófagos, actividad enzimática, β-galactosidasa, β-glucuronidasa, β-hexosaminidasa, enfermedades de depósito lisosomal, protocolos, Lysosome, fibroblasts, leukocytes, monocytes, macrophages, enzyme activity, β-galactosidase, β-glucuronidase, β-hexosaminidase, lysosomal storage diseases, protocols, Biología - Tesis y disertaciones académicas, Lisosomas
وصف الملف: PDF; application/pdf
العلاقة: http://hdl.handle.net/10554/66226Test; instname:Pontificia Universidad Javeriana; reponame:Repositorio Institucional - Pontificia Universidad Javeriana; repourl:https://repository.javeriana.edu.coTest
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5رسالة جامعية
المؤلفون: Gray, James Andrew Russell
المساهمون: Platt, Frances
مصطلحات موضوعية: 616.3, Heat shock proteins, Lysosomal storage diseases, Central nervous system--Abnormalities
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6رسالة جامعية
المؤلفون: Chen, Chun-Wu
المساهمون: Platt, Fran
مصطلحات موضوعية: 616.7, Pharmacology, Lysosomal storage diseases, metal, biomarker, glycosphingolipids
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7رسالة جامعية
المؤلفون: Tkalčić, Magdalena
المساهمون: Matulić, Maja
مصطلحات موضوعية: lizosom, mukolipidoza II, nasljedne bolesti, lizosomske bolesti nakupljanja, metabolizam, lysosome, mucolipidosis II, hereditary diseases, lysosomal storage diseases, metabolism, PRIRODNE ZNANOSTI. Biologija, NATURAL SCIENCES. Biology
وصف الملف: application/pdf
العلاقة: https://zir.nsk.hr/islandora/object/pmf:11999Test; https://urn.nsk.hr/urn:nbn:hr:217:400519Test; https://repozitorij.unizg.hr/islandora/object/pmf:11999Test; https://repozitorij.unizg.hr/islandora/object/pmf:11999/datastream/PDFTest
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8رسالة جامعية
المؤلفون: Dornelles, Alícia Dorneles
المساهمون: Schwartz, Ida Vanessa Doederlein, Oliveira Junior, Haliton Alves de
مصطلحات موضوعية: Terapia de reposição de enzimas, Doença de depósito de glicogênio tipo II, Revisão sistemática, Metanálise, Erros inatos do metabolismo, Enzyme replacement therapy, Pompe disease, Glycogen storage disease type II, Systematic review, Meta-analysis, Inborn errors of metabolism, Lysosomal storage diseases
وصف الملف: application/pdf
العلاقة: http://hdl.handle.net/10183/252566Test; 001154130
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9رسالة جامعية
المؤلفون: Olarte Avellaneda, Sergio
المساهمون: Alméciga Díaz, Carlos Javier, Salazar Pulido, Luz Mary, Instituto de Errores Innatos del Metabolismo
مصطلحات موضوعية: 610 - Medicina y salud::615 - Farmacología y terapéutica, Química Farmacéutica, Mucopolisacaridosis, Enfermedades por Almacenamiento Lisosomal, Chemistry, Pharmaceutical, Mucopolysaccharidoses, Lysosomal Storage Diseases, MPS IVA, GALNS, Queratán sulfato, Chaperonas farmacológicas, Acoplamiento molecular computacional, Dinámica molecular, Tamizaje virtual, Keratan sulfate, Pharmacological chaperones, Computational molecular docking, Molecular dynamic, Virtual screening
وصف الملف: 125 páginas; application/pdf
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Drugs. 2019;79(10):1103-34. Epub 2019/06/19.; Parenti G. Treating lysosomal storage diseases with pharmacological chaperones: from concept to clinics. EMBO Mol Med. 2009;1(5):268-79. Epub 2010/01/06.; Olarte-Avellaneda S, Rodriguez-Lopez A, Almeciga-Diaz CJ, Barrera LA. Computational analysis of human N-acetylgalactosamine-6-sulfate sulfatase enzyme: an update in genotype-phenotype correlation for Morquio A. Mol Biol Rep. 2014;41(11):7073-88. Epub 2014/10/08.; Rivera-Colon Y, Schutsky EK, Kita AZ, Garman SC. The Structure of Human GALNS Reveals the Molecular Basis for Mucopolysaccharidosis IV A. J Mol Biol. 2012. Epub 2012/09/04.; Olarte-Avellaneda S, Rodríguez-López A, Alméciga-Díaz CJ. In-silico analysis of the active cavity of N-acetylgalactosamine-6-sulfate sulfatase in eight species. In: Castillo LF, Cristancho M, Isaza G, Pinzón A, Corchado JM, editors. Advances in Computational Biology: Springer International Publishing; 2014. p. 141-6.; Almeciga-Diaz CJ, Hidalgo OA, Olarte-Avellaneda S, Rodriguez-Lopez A, Guzman E, Garzon R, et al. Identification of ezetimibe and pranlukast as pharmacological chaperones for treatment of the rare disease Mucopolysaccharidosis type IVA. J Med Chem. 2019. Epub 2019/06/13.; Losada Díaz JC, Cepeda del Castillo J, Rodriguez-López EA, Alméciga-Díaz CJ. Advances in the development of pharmacological chaperones for the mucopolysaccharidoses. Int J Mol Sci. 2020;21(1):232.; Montano AM, Tomatsu S, Gottesman GS, Smith M, Orii T. International Morquio A Registry: clinical manifestation and natural course of Morquio A disease. J Inherit Metab Dis. 2007;30(2):165-74. Epub 2007/03/10.; Tomatsu S, Montano AM, Nishioka T, Gutierrez MA, Pena OM, Tranda Firescu GG, et al. Mutation and polymorphism spectrum of the GALNS gene in mucopolysaccharidosis IVA (Morquio A). Hum Mutat. 2005;26(6):500-12. 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Clinical and molecular characteristics of colombian patients with mucopolysaccharidosis IVA, and description of a new galns gene mutation. Mol Genet Metab Rep. 2018;16:53-6. Epub 2018/08/11.; Gómez A, Robles R, Suárez-Obando F. Estimation of the mucopolysaccharidoses frequencies and cluster analysis in the Colombian provinces of Cundinamarca and Boyacá. Biomedica : revista del Instituto Nacional de Salud. 2012;32:602-9.; Leadley RM, Lang S, Misso K, Bekkering T, Ross J, Akiyama T, et al. A systematic review of the prevalence of Morquio A syndrome: challenges for study reporting in rare diseases. Orphanet J Rare Dis. 2014;9:173. Epub 2014/11/19.; Bernal J, Briceño I. Genetic and other diseases in the pottery of Tumaco-La Tolita culture in Colombia–Ecuador. Clin Genet. 2006;70:188-91.; Pachajoa H, Rodriguez CA, Isaza C. Possible case of Morquio syndrome in the pottery of Tumaco-Tolita culture. Rev Neurol. 2009;48(1):52. Epub 2009/01/16. Posible caso de sindrome de Morquio en la ceramica de la cultura tumaco-tolita.; Tomatsu S, Sawamoto K, Shimada T, Bober MB, Kubaski F, Yasuda E, et al. Enzyme replacement therapy for treating mucopolysaccharidosis type IVA (Morquio A syndrome): effect and limitations. Expert Opin Orphan Drugs. 2015;3(11):1279-90.; Sáenz H, Lareo L, Poutou RA, Sosa AC, Barrera LA. Predicción computacional de la estructura terciaria de la iduronato 2-sulfato sulfatasa. Biomedica. 2007;27(001):7-20.; Echeverri OY, Guevara JM, Espejo-Mojica AJ, Ardila A, Pulido N, Reyes M, et al. Research, diagnosis and education in inborn errors of metabolism in Colombia: 20 years' experience from a reference center. Orphanet J Rare Dis. 2018;13(1):141. Epub 2018/08/18.; Rodriguez A, Espejo AJ, Hernandez A, Velasquez OL, Lizaraso LM, Cordoba HA, et al. Enzyme replacement therapy for Morquio A: an active recombinant N-acetylgalactosamine-6-sulfate sulfatase produced in Escherichia coli BL21. J Ind Microbiol Biotechnol. 2010;37(11):1193-201. Epub 2010/06/29.; Mosquera A, Rodríguez A, Soto C, Leonardi F, Espejo A, Sánchez OF, et al. Characterization of a recombinant N-acetylgalactosamine-6-sulfate sulfatase produced in E. coli for enzyme replacement therapy of Morquio A disease. Process Biochemistry. 2012:1-6.; Hernandez A, Velasquez O, Leonardi F, Soto C, Rodriguez A, Lizaraso L, et al. Effect of Culture Conditions and Signal Peptide on Production of Human Recombinant N-Acetylgalactosamine-6-Sulfate Sulfatase in Escherichia coli BL21. J Microbiol Biotechnol. 2013;23(5):689-98. Epub 2013/05/08.; Reyes LH, Cardona C, Pimentel L, Rodriguez-Lopez A, Almeciga-Diaz CJ. Improvement in the production of the human recombinant enzyme N-acetylgalactosamine-6-sulfatase (rhGALNS) in Escherichia coli using synthetic biology approaches. Scientific reports. 2017;7(1):5844. Epub 2017/07/21.; Rodriguez-Lopez A, Almeciga-Diaz CJ, Sanchez J, Moreno J, Beltran L, Diaz D, et al. Recombinant human N-acetylgalactosamine-6-sulfate sulfatase (GALNS) produced in the methylotrophic yeast Pichia pastoris. Sci Rep. 2016;6:29329. Epub 2016/07/06.; Alméciga-Diaz CJ, A. R-PM, Echeverri OY, Montaño AM, Tomatsu S, Barrera LA. Uso de vectores derivados de virus adenoasociados para el tratamiento de la enfermedad de Morquio A. Univ Med. 2009;50(3):356-79.; Almeciga-Diaz CJ, Montano AM, Tomatsu S, Barrera LA. Adeno-associated virus gene transfer in Morquio A disease - effect of promoters and sulfatase-modifying factor 1. FEBS J. 2010;277(17):3608-19. Epub 2010/08/19.; Almeciga-Diaz CJ, Rueda-Paramo MA, Espejo AJ, Echeverri OY, Montano A, Tomatsu S, et al. Effect of elongation factor 1alpha promoter and SUMF1 over in vitro expression of N-acetylgalactosamine-6-sulfate sulfatase. Mol Biol Rep. 2009;36(7):1863-70. Epub 2008/11/08.; Gutierrez MA, Vallejo F, Tomatsu S, Cerón F, Almeciga-Diaz CJ, Dominguez MC, et al. Construcción de un vector de expresión derivado de virus adenoasociados para corregir In vitro el defecto genético de la enfermedad de Morquio A. Biomedica. 2008;28(003):448-59.; Almeciga C, Cuaspa R, Herrera J, Barbosa H, Barrera L. New viral vectors for Morquio syndrome type A gene therapy. Molecular Genetics and Metabolism. 2013;108(2):19.; Alméciga-Díaz CJ MA, Barrera LA., Tomatsu S. . Tailoring the AAV2 capsid vector for bone-targeting. . Pediatr Res 2018.; Alméciga-Diaz CJ, Barrera LA. Design and applications of gene therapy vectors for mucopolysaccharidosis in Colombia. Gene Therapy. 2019.; Alméciga-Diaz C, Alexander R. A system biology view of the glycosaminoglycans degradation pathways. Molecular Genetics and Metabolism. 2012.; Salazar DA, Rodriguez-Lopez A, Herreno A, Barbosa H, Herrera J, Ardila A, et al. Systems biology study of mucopolysaccharidosis using a human metabolic reconstruction network. Mol Genet Metab. 2016;117(2):129-39. 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Tesis de grado Universidad Austral de Chile 2016; https://repositorio.unal.edu.co/handle/unal/82006Test; Universidad Nacional de Colombia; Repositorio Institucional Universidad Nacional de Colombia; https://repositorio.unal.edu.coTest/
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10رسالة جامعية
المساهمون: Macedo, Maria de Fátima Matos Almeida Henriques
مصطلحات موضوعية: Lysosomal storage diseases, Lipid antigen presentation, CD1b, CD1d, Dendritic cells, Monocytes, Macrophages, Natural killer T cells
العلاقة: SFRH/BD/113107/2015; Norte-01-0145-FEDER-000012; http://hdl.handle.net/10773/30452Test; 101584610
