12 datasets found
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Federal
Taking stock of gene therapy for cystic fibrosis
U.S. Department of Health & Human Services —
The identification of the cystic fibrosis (CF) gene opened the way for gene therapy. In the ten years since then, proof of principle in vitro and then in animal... -
Federal
The PEST sequence does not contribute to the stability of the cystic fibrosis transmembrane conductance regulator
U.S. Department of Health & Human Services —
Background Endoplasmic reticulum retention of misfolded cystic fibrosis transmembrane conductance regulator (CFTR) mutants and their rapid degradation is the major... -
Federal
"Bronchial Artery Delivery of Viral Vectors for Gene delivery in Cystic Fibrosis; Superior to Airway Delivery?"
U.S. Department of Health & Human Services —
Background Attempts at gene therapy for the pulmonary manifestations of Cystic Fibrosis have relied mainly on airway delivery. However the efficiency of gene transfer... -
Federal
The role of secretory leukocyte proteinase inhibitor and elafin (elastase-specific inhibitor/skin-derived antileukoprotease) as alarm antiproteinases in inflammatory lung disease
U.S. Department of Health & Human Services —
Secretory leukocyte proteinase inhibitor and elafin are two low-molecular-mass elastase inhibitors that are mainly synthesized locally at mucosal sites. It is thought... -
Federal
Ileal mucosal bile acid absorption is increased in Cftr knockout mice
U.S. Department of Health & Human Services —
Background Excessive loss of bile acids in stool has been reported in patients with cystic fibrosis. Some data suggest that a defect in mucosal bile acid transport... -
Federal
Research Article: BMC Medical Genetics
U.S. Department of Health & Human Services —
Background SFHR (small fragment homologous replacement)-mediated targeting is a process that has been used to correct specific mutations in mammalian cells. This... -
Federal
Size does matter: overcoming the adeno-associated virus packaging limit
U.S. Department of Health & Human Services —
Recombinant adeno-associated virus (rAAV) vectors mediate long-term gene transfer without any known toxicity. The primary limitation of rAAV has been the small size... -
Federal
Unique presentations and chronic complications in adult cystic fibrosis: do they teach us anything about CFTR?
U.S. Department of Health & Human Services —
The increase in numbers of adults with cystic fibrosis (CF) has allowed us to identify previously unrecognized chronic complications of CF, as well as appreciate... -
Federal
Glycosylation and the cystic fibrosis transmembrane conductance regulator
U.S. Department of Health & Human Services —
The cystic fibrosis transmembrane conductance regulator (CFTR) has been known for the past 11 years to be a membrane glycoprotein with chloride channel activity. Only... -
Federal
'CFTR-opathies': disease phenotypes associated with cystic fibrosis transmembrane regulator gene mutations
U.S. Department of Health & Human Services —
Cystic fibrosis is a genetic disease that is associated with abnormal sweat electrolytes, sino-pulmonary disease, exocrine pancreatic insufficiency, and male... -
Federal
Analysis of transcriptomic data from duodena of mice exposed to hexavalent chromium in drinking water: Supplemental data supporting a research report.
U.S. Environmental Protection Agency —
These secondary data have been produced from the gene expression data generated from duodena of mice exposed to hexavalent chromium in drinking water and deposited in... -
Federal
Severe reperfusion lung injury after double lung transplantation
U.S. Department of Health & Human Services —
Aim To demonstrate the effects of combined inhaled nitric oxide and surfactant replacement as treatment for acute respiratory distress syndrome. This treatment has...
12 datasets found