Modely ex vivo vedoucí k personalizaci diagnostického a terapeutického přístupu u pacientů s cystickou fibrózou

• Název práce v češtině: Modely ex vivo vedoucí k personalizaci diagnostického a terapeutického přístupu u pacientů s cystickou fibrózou
• Název v anglickém jazyce: Ex vivo models leading to personalized diagnostic and therapeutic approach in patient with cystic fibrosis
• Klíčová slova: Cystická fibróza, tkáňové modely, střevní organoidy, CFTR modulátory, personalizovaná medicína
• Klíčová slova anglicky: Cystic fibrosis, tissue models, intestinal organoids, CFTR modulators, personalised medicine
• Akademický rok vypsání: 2017/2018
• Typ práce: disertační práce
• Jazyk práce: čeština
• Ústav: Ústav lékařské mikrobiologie (13-323)
• Vedoucí / školitel: prof. MUDr. Pavel Dřevínek, Ph.D.
• Řešitel: skrytý - zadáno a potvrzeno stud. odd.
• Datum přihlášení: 07.10.2017
• Datum zadání: 07.10.2017
• Datum potvrzení stud. oddělením: 07.10.2017
• Datum a čas obhajoby: 26.09.2024 10:00
• Datum odevzdání elektronické podoby: 25.06.2024
• Datum odevzdání tištěné podoby: 25.06.2024
• Datum proběhlé obhajoby: 26.09.2024
• Oponenti: MUDr. Renata Gaillyová, Ph.D.

Seznam odborné literatury

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• Bobadilla, J.L. et al. (2002) ‘Cystic fibrosis: A worldwide analysis of CFTR mutations - Correlation with incidence data and application to screening’, Human Mutation, pp. 575–606. doi: 10.1002/humu.10041.

• de Boeck, K. et al. (2014) ‘The relative frequency of CFTR mutation classes in European patients with cystic fibrosis’, Journal of Cystic Fibrosis, 13(4), pp. 403–409. doi: 10.1016/j.jcf.2013.12.003.

• de Boeck, K. and Amaral, M.D. (2016) ‘Progress in therapies for cystic fibrosis’, The Lancet Respiratory Medicine. Lancet Publishing Group, pp. 662–674. doi: 10.1016/S2213-2600(16)00023-0.

• Clancy, J.P. et al. (2019) ‘CFTR modulator theratyping: Current status, gaps and future directions’, Journal of Cystic Fibrosis. Elsevier B.V., pp. 22–34. doi: 10.1016/j.jcf.2018.05.004.

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• Dekkers, J.F., van der Ent, C.K. and Beekman, J.M. (2013) ‘Novel opportunities for CFTR-targeting drug development using organoids’, Rare Diseases, 1(1), p. e27112. doi: 10.4161/rdis.27112.

• Heijerman, H.G.M. et al. (2019) ‘Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial’, The Lancet, 394(10212), pp. 1940–1948. doi: 10.1016/S0140-6736(19)32597-8.

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• Veit, G. et al. (2016) ‘From CFTR biology toward combinatorial pharmacotherapy: Expanded classification of cystic fibrosis mutations’, Molecular Biology of the Cell, 27(3), pp. 424–433. doi: 10.1091/mbc.E14-04-0935.

• Zhang, Z. and Chen, J. (2016) ‘Atomic Structure of the Cystic Fibrosis Transmembrane Conductance Regulator’, Cell, 167(6), pp. 1586-1597.e9. doi: 10.1016/j.cell.2016.11.014.