Paving the way for new CF therapies
Even before any line of research can yield any preliminary data, it is important that scientists create the appropriate tools with which they can carry out their intended tasks. In cystic fibrosis (CF), disease onset is attributed to a structurally deficient mutated protein, which under normal circumstances acts as a chloride ion channel in the cell membrane. This protein has been termed the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) and it essentially controls the entrance and exit of chloride ions to and from the cell. Non- or partially-functioning CFTR molecules can cause serious cell damage, which manifests as a variety of symptoms in CF. Attempting to rescue these cells relies upon the discovery and optimisation of novel compounds which can affect CFTR's function. However, before such compounds can be sought, it is crucial to create animal models which can mimic CF pathology. In other words, the aim is to breed laboratory animals which carry the mutated form(s) of CFTR and retain this phenotype through subsequent generations. The Erasmus University Rotterdam undertook to breed specific strains of mice, which carry a specific mutated version of CFTR. The genetic constructs underwent a series of tests aimed at determining the suitability of the model and the rate of disease progression. A number of these models was deemed suitable and they have since been used for the testing of novel therapeutic approaches. These approaches include genetic as well as pharmaceutical intervention methods. The animal models described herein could form the basis for CF projects throughout the pharmaceutical industry and academia.
