Patient-Derived Organoids for Personalized Medicine in Cystic Fibrosis

Speelman, Leanne (2024) Patient-Derived Organoids for Personalized Medicine in Cystic Fibrosis. Bachelor's Thesis, Life Science and Technology.

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Abstract

CF is a life-threatening disease caused by mutations in the CFTR gene. The development of CFTR modulator drugs has majorly improved CF treatment, but not all patients are eligible for these drugs. This is due to the large variety of clinical phenotypes resulting from over 700 identified disease-causing CFTR mutations. Currently,10 to 20% of CF patients are non-eligible for highly effective modulator treatments, as these patients carry rare CFTR mutations and have therefore often been excluded from clinical trials. To increase the accessibility to CFTR-restoring therapies for these patients, personalized medicine approaches could provide an outcome. Patient-derived organoids (PDOs) have shown to be a promising tool for this in a cost-efficient and patient-friendly manner. A forskolin-induced swelling assay can reliably assess CFTR function in patient-derived intestinal organoids and therefore paved the way for organoid usage in CF treatment. Currently, PDOs have shown to possess great potential in identifying efficient therapies for individual CF patients. Moreover, organoids can be used in the early developmental stages of new CF therapies specified for subgroups of CF patients with rare mutations. Biobanks that store these organoids can further expand the possibilities of PDO use in CF research and personalized treatments. This thesis provides an overview of current CF therapies, developments in PDOs, and the usage and potential of PDOs for personalized medicine in CF.

Item Type:	Thesis (Bachelor's Thesis)
Supervisor name:	Coppes, R.P.
Degree programme:	Life Science and Technology
Thesis type:	Bachelor's Thesis
Language:	English
Date Deposited:	09 Jul 2024 09:26
Last Modified:	09 Jul 2024 09:26
URI:	https://fse.studenttheses.ub.rug.nl/id/eprint/33161

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