Organoid cultures, mini-organs able to reflect the complex cell biology of a patient’s own tissue, are a hot topic in cystic fibrosis.
Cystic fibrosis (CF) is caused by mutations in a single gene (the CFTR gene), but different mutations have different functional consequences on the CFTR protein. Mutation-specific CFTR correctors are drugs designed to correct faults associated with specific mutations. However, there are approximately 2,000 different CFTR mutations and therefore difficult to assess which drug/s might be required for individual patients. Patient-derived organoid cultures can fill this gap. Organoid cultures are mini-organs with a stable genotype and phenotype and therefore accurately reflect the complex cell biology of the patient’s own tissue. In CF patients, organoid cultures from rectal biopsies express the dysfunctional CFTR gene and protein. Culture of CF organoids in the presence of different mutation-specific CFTR corrector drugs can be performed to determine which drug, or combination of drugs, might work for an individual patient or groups of patients with a given mutation. The unique ability of organoid cultures to stably recapitulate the patients’ own diseased tissues in vitro means they have wide application for the understanding and treatment of CF and many different disease types.
—Mary McElroy, Principal Scientist, In Vivo Discovery/Cell Therapy