Personalised medicine for cystic fibrosis: treating the basic defect

The concept of personalised or stratified therapies is not a new one. For centuries, physicians have observed that the manifestation of a disease and its response to intervention can vary according to many factors including age, sex, ethnicity, diet and the type of administered drug [1]. Hippocrates, for example, observed over 2,000 years ago that patients can respond very differently to various medications [2]. It wasn’t until 1998, however, that the term “personalised therapy” was first used [3]. This can be defined as a therapy prescribed using molecular profiling technologies to tailor the right therapeutic strategy for the right person at the right time. The therapy is typically accompanied by a “companion test” or clinical biomarker to identify responders whilst…