Current Drug Targets - Volume 16, Issue 9, 2015

Cystic fibrosis (CF) is a lethal autosomal recessive disease that causes severe damage to the respiratory and digestive systems. It results from a dysfunctional CF Transmembrane Conductance Regulator (CFTR) protein, which is a cAMP- regulated epithelial chloride channel. CFTR is also a subtype of the ABC-transporter superfamily, and is expressed primarily in the apical membrane of epithelial cells in the airways, pancre Read More

PDZ domains play an essential role in a number of cellular processes by facilitating protein scaffolding and assembly of protein complexes. These domains consist of 80 to 90 amino acids and are found to recognize short C-terminal sequences of target proteins. Protein complex formation between PDZ target molecules can lead to a number of signaling and regulatory cascades that may either promote or inhibit the activat Read More

Cystic fibrosis (CF) is the most common life shortening autosomal inherited disorder, affecting 1 in 2500 newborns in the Caucasian population. In CF the lung pathology is associated with dehydration of the airways epithelial surface which in part results from Na+ hyperabsorption via the epithelial sodium channel (ENaC). The molecular mechanisms of this Na+ hyperabsorption and its correlation with the underlying geneti Read More

Cystic Fibrosis (CF) is largely caused by protein misfolding and the loss of function of a plasma membrane anion channel known as the cystic fibrosis transmembrane conductance regulator (CFTR). The most common CF-causing mutation, F508del, leads to severe conformational defect in CFTR. The cellular chaperone machinery plays an important role in CFTR biogenesis and quality control. Multiple attempts have been made to i Read More

The SUMOylation pathway is involved in the regulation of numerous and diverse cellular functions, nuclear as well as extra-nuclear. Thus, it is not surprising that SUMO pathway components are implicated in diseases as diverse as cystic fibrosis, cancer and neurodegenerative diseases. Therefore, the components of the SUMOylation pathway should provide valid therapeutic targets for manipulation. While the related ubi Read More

Cystic fibrosis (CF) is a deadly genetic disease that affects the lungs and digestive system. A mutation in the CF transmembrane conductance regulator (CFTR) gene is the cause of the disease. How epigenetics contributes to CFTR expression is still poorly understood. Epigenetics is a mechanism that alters gene expression without changing the underlying DNA sequence. Epigenetic mechanisms include DNA methylatio Read More

The formation of competent spermatozoa is associated with the movement of large quantities of water and electrolytes in the various tissues and luminal fluids of the male reproductive tract. The cystic fibrosis transmembrane conductance regulator (CFTR) is a cAMP-activated Cl− and HCO3 − membrane transporter. CFTR gene mutations cause cystic fibrosis (CF), the most common lethal genetic disease in Caucasians. Of note, Read More

Personalized drug therapy for cystic fibrosis (CF) is a long-term dream for CF patients, caregivers, physicians and researchers. After years of study, the fiction of personalized treatment has turned to hope. Basic information about CFTR mutations classes and new treatments is needed if we are to deal properly with the new CF era. The problems involved in this issue, however, should be evaluated with greater care and attention. Read More