The gene associated with cystic fibrosis (CF) encodes a membrane-associated, N-linked
glycoprotein called CFTR. Mutations wem introduced into CFTR at residues known to be …

The primary cause of Huntington's disease (HD) is expression of huntingtin with a
polyglutamine expansion. Despite an absence of consensus on the mechanism(s) of toxicity, …

CFTR, the protein associated with cystic fibrosis, is phosphorylated on serine residues in
response to CAMP agonists. Serines 660, 737, 795, and 613 were identified as in vivo targets …

The cystic fibrosis transmembrane conductance regulator (CFTR) was expressed in cultured
cystic fibrosis airway epithelial cells and Cl − channel activation assessed in single cells …

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder caused
by mutations in the SMN1 gene that result in a deficiency of SMN protein. One approach to …

Background Lung delivery of plasmid DNA encoding the CFTR gene complexed with a
cationic liposome is a potential treatment option for patients with cystic fibrosis. We aimed to …

Cationic lipid-mediated gene transfer of cystic fibrosis transmembrane conductance regulator
(CFTR) cDNA represents a promising approach for treatment of cystic fibrosis (CF). Here, …

Antisense oligonucleotides (ASOs) hold promise for gene-specific knockdown in diseases
that involve RNA or protein gain-of-function effects. In the hereditary degenerative disease …

Hypoxia-inducible factor-1 (HIF-1) mediates transcriptional activation of vascular endothelial
growth factor (VEGF) and other hypoxia-responsive genes. Transgenic expression of a …

CYSTIC fibrosis (CF) is a common lethal genetic disease that manifests itself in airway and
other epithelial cells as defective chloride ion absorption and secretion 1,2 , resulting at least …