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Therapeutic strategies in cystic fibrosis
Křesťan, Jan ; Bořek Dohalská, Lucie (advisor) ; Hudeček, Jiří (referee)
Cystic fibrosis is an autozomal recessive disease caused by mutations in the CFTR gene. The aim of this work is conclusion of therecent knowledgefrom theusage of CFTR modulators in the treatment of cystic fibrosis. The first part of this bachelor thesis is dedicated to the disease itself, its history and symptoms. In the form of description of the CFTR gene and its mutations which lead to the cystic fibrosis disease, the causes of the disease are described. Function of CFTR protein is also mentioned. The second part of this thesis is focused on conclusion of the current knowledge of the CFTR modulators treatment. In the last chapter of the work, possible future changes and ways of treating patients with CFTR modulators therapy are considered.
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The development of a model system for studying chloride ion transport in the epithelial cells of patients with cystic fibrosis
Pecková, Kateřina ; Bořek Dohalská, Lucie (advisor) ; Kubíčková, Božena (referee)
Cystic fibrosis is caused by a genetic defect in the CFTR protein, whose main function is chloride transport across epithelial cells. The measurement of CFTR ability to transport chloride is considered a good, and perhaps, the only practical method to assess its activity. In this thesis, the transport of chloride ions across the CFTR channel was studied using airway epithelial cell lines of healthy patients (NuLi-1) and patients with cystic fibrosis (CuFi-1). A fluorescent method using a fluorescent chloride-sensitive probe N-(ethoxycarbonylmethyl)-6-methoxyquinolinium (MQAE) was chosen and optimized. This compound is providing fluorescence in the blue part of the spectrum and has the greatest sensitivity to chloride ions. In the development of an optimal method two approaches of chloride transport measurement were used. In the first experiment the secretion of the chloride ions to the buffer containing MQAE was measured. In the second one the dye had to be loaded into cells before performing experiment. Then, the MQAE fluorescence quenched by intracellular chloride was monitored by a change in the fluorescence intensity of the probe. The second method was considered as a usefull and more reproducible to study chloride transport across cell membranes. Moreover, the influence of the CFTR modulator...
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Therapeutic strategies in cystic fibrosis
Křesťan, Jan ; Bořek Dohalská, Lucie (advisor) ; Hudeček, Jiří (referee)
Cystic fibrosis is an autozomal recessive disease caused by mutations in the CFTR gene. The aim of this work is conclusion of therecent knowledgefrom theusage of CFTR modulators in the treatment of cystic fibrosis. The first part of this bachelor thesis is dedicated to the disease itself, its history and symptoms. In the form of description of the CFTR gene and its mutations which lead to the cystic fibrosis disease, the causes of the disease are described. Function of CFTR protein is also mentioned. The second part of this thesis is focused on conclusion of the current knowledge of the CFTR modulators treatment. In the last chapter of the work, possible future changes and ways of treating patients with CFTR modulators therapy are considered.
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The development of a model system for studying chloride ion transport in the epithelial cells of patients with cystic fibrosis
Pecková, Kateřina ; Bořek Dohalská, Lucie (advisor) ; Kubíčková, Božena (referee)
Cystic fibrosis is caused by a genetic defect in the CFTR protein, whose main function is chloride transport across epithelial cells. The measurement of CFTR ability to transport chloride is considered a good, and perhaps, the only practical method to assess its activity. In this thesis, the transport of chloride ions across the CFTR channel was studied using airway epithelial cell lines of healthy patients (NuLi-1) and patients with cystic fibrosis (CuFi-1). A fluorescent method using a fluorescent chloride-sensitive probe N-(ethoxycarbonylmethyl)-6-methoxyquinolinium (MQAE) was chosen and optimized. This compound is providing fluorescence in the blue part of the spectrum and has the greatest sensitivity to chloride ions. In the development of an optimal method two approaches of chloride transport measurement were used. In the first experiment the secretion of the chloride ions to the buffer containing MQAE was measured. In the second one the dye had to be loaded into cells before performing experiment. Then, the MQAE fluorescence quenched by intracellular chloride was monitored by a change in the fluorescence intensity of the probe. The second method was considered as a usefull and more reproducible to study chloride transport across cell membranes. Moreover, the influence of the CFTR modulator...
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