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Despite being a multi\u2010organ disease, <jats:styled-content style=\"fixed-case\">CF<\/jats:styled-content> is classically diagnosed by symptoms of acute\/chronic respiratory disease, with persistent pulmonary infections and mucus plugging of the airways and failure to thrive. These multiple symptoms originate from dysfunction of the <jats:styled-content style=\"fixed-case\">CF<\/jats:styled-content> transmembrane conductance regulator (<jats:styled-content style=\"fixed-case\">CFTR<\/jats:styled-content>) protein, a channel that mediates anion transport across epithelia. Indeed, establishment of a definite <jats:styled-content style=\"fixed-case\">CF<\/jats:styled-content> diagnosis requires proof of <jats:styled-content style=\"fixed-case\">CFTR<\/jats:styled-content> dysfunction, commonly through the so\u2010called sweat Cl<jats:sup>\u2212<\/jats:sup> test. Many drug therapies, including mucolytics and antibiotics, aim to alleviate the symptoms of <jats:styled-content style=\"fixed-case\">CF<\/jats:styled-content> lung disease. However, new therapies to modulate defective <jats:styled-content style=\"fixed-case\">CFTR<\/jats:styled-content>, the basic defect underlying <jats:styled-content style=\"fixed-case\">CF<\/jats:styled-content>, have started to reach the clinic, and several others are in development or in clinical trials. The novelty of these therapies is that, besides targeting the basic defect underlying <jats:styled-content style=\"fixed-case\">CF<\/jats:styled-content>, they are mutation specific. Indeed, even this monogenic disease is influenced by a large number of different genes and biological pathways as well as by environmental factors that are difficult to assess. Accordingly, every person with <jats:styled-content style=\"fixed-case\">CF<\/jats:styled-content> is unique and so functional assessment of patients' tissues <jats:italic>ex vivo<\/jats:italic> is key for diagnosing and predicting the severity of this disease. Of note, such assessment will also be crucial to assess drug responses, in order to effectively treat all <jats:styled-content style=\"fixed-case\">CF<\/jats:styled-content> patients. It is not because it is a monogenic disorder that personalized treatment for <jats:styled-content style=\"fixed-case\">CF<\/jats:styled-content> is much easier than for complex disorders.<\/jats:p>","DOI":"10.1111\/joim.12314","type":"journal-article","created":{"date-parts":[[2014,9,29]],"date-time":"2014-09-29T21:17:42Z","timestamp":1412025462000},"page":"155-166","source":"Crossref","is-referenced-by-count":101,"title":["Novel personalized therapies for cystic fibrosis: treating the basic defect in all patients"],"prefix":"10.1111","volume":"277","author":[{"given":"M. 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