Wilfried Dalemans

TL;DR: Observations suggest the feasibility of in vivo CFTR gene transfer as therapy for the pulmonary manifestations of CF.

TL;DR: The ΔF508 mutation seems to have two major consequences, an abnormal translocation of the CFTR protein which limits membrane insertion, and an abnormal function in mediating Cl- transport.

TL;DR: Analysis of the CFTR gene promoter suggests it is a housekeeping gene, implying more widespread expression than only in epithelial cells, and the presence of CFTR mRNA transcripts in all cells evaluated except HL-60 cells.

TL;DR: Findings are consistent with the concept that the CFTR delta Phe 508 mutation modifies the intracellular maturation and trafficking of the protein, leading to an altered subcellular distribution of the delta P he 508 mutant CFTR.

TL;DR: By crossing transgenic mice synthesizing FIX with others prone to develop hepatoma, progeny which co‐express the transgenes in hepatocytes were obtained and Hepatoma‐derived cell lines were shown to have a differentiated phenotype and secrete active human FIX for many generations.