It is extremely rare for a single experiment to be so impactful and timely that it shapes and
forecasts the experiments of the next decade. Here, we review how two such experiments …

The discovery of the Cystic fibrosis (CF) gene in 1989 has paved the way for incredible
progress in treating the disease such that the mean survival age of individuals living with CF …

Classification of CFTR mutation classes. - Abstract - Europe PMC Sign in | Create an account
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Since the early days of its conceptualization and application, human gene transfer held the
promise of a permanent solution to genetic diseases including cystic fibrosis (CF). This field …

Cystic fibrosis (CF) is a lethal autosomal recessive disease caused by mutations in the cystic
fibrosis transmembrane conductance regulator (CFTR) gene. Nuclease-mediated precise …

There is a strong rationale to consider future cell therapeutic approaches for cystic fibrosis
(CF) in which autologous proximal airway basal stem cells, corrected for CFTR mutations …

The advent of the knowledge on human genetics, by the identification of disease-associated
variants, culminated in the understanding of human variability. With the genetic knowledge …

Cystic Fibrosis (CF), an autosomal recessive genetic disease, is caused by a mutation in the
gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR). This …

Cystic fibrosis (CF) is the main genetic cause of death among the Caucasian population.
The disease is characterized by abnormal fluid and electrolyte mobility across secretory …

Recent advances in genome editing with programmable nucleases have opened up new
avenues for multiple applications, from basic research to clinical therapy. The ease of use of …