Vascular disease is still the leading cause of morbidity and mortality in the Western world,
and the primary cause of myocardial infarction, stroke, and ischemia. The biology of vascular …

The advent of techniques such as microarrays and high-throughput sequencing has
revolutionized our ability to examine messenger RNA (mRNA) expression within the …

Chronic respiratory diseases encompass a group of diverse conditions affecting the airways,
which all impair lung function over time. They include cystic fibrosis (CF), idiopathic …

Chronic lung disease has become a leading cause of death in recent years. Despite several
attempts to discover and develop new therapeutic approaches, patients often suffer a poor …

Highlights•Next-generation sequencing technology accelerates rare disease research.•
Accumulated genomics information aids orphan drug development.•Novel drug …

Since the discovery and classification of non-coding RNAs, their roles have gained great
attention. In this respect, microRNAs and long non-coding RNAs have been firmly …

MicroRNAs that are overexpressed in cystic fibrosis (CF) bronchial epithelial cells (BEC)
negatively regulate CFTR and nullify the beneficial effects of CFTR modulators. We …

Congenital diarrhoeal disorders (CDDs) represent an evolving web of rare chronic
enteropathies, with a typical onset early in life. In many of these conditions, severe chronic …

Background In recent years, patients with cystic fibrosis (CF) conductance regulator (CFTR)
variant poly (T) sequences have been increasingly reported with a wide spectrum of clinical …

Lung diseases are one of the leading causes of mortality and morbidity worldwide and
effective therapies are imperfect. Nonetheless, recently some novel strategies have been …