Abstract: The present invention discloses a therapeutic target for the treatment of cystic fibrosis. It was found that inhibition of non-lysosomal glucosylceramidase (GBA2) sufficiently restores chloride current in cells from CF patients carrying the common delF508-CFTR mutation. With the catalytic center (4) of the enzyme positioned on top of the membrane bilayer face particularly potent inhibitors are found in deoxynojirimycin derivatives having a group that is capable of inserting in the membrane bilayer.

Abstract: The present invention discloses a therapeutic target for the treatment of cystic fibrosis. It was found that inhibition of non-lysosomal glucosylceramidase (GBA2) sufficiently restores chloride current in cells from CF patients carrying the common delF508-CFTR mutation. With the catalytic centre (4) of the enzyme positioned on top of the membrane bilayer face particularly potent inhibitors are found in deoxynojirimycin derivatives having a group that is capable of inserting in the membrane bilayer.

Abstract: The invention provides a mammalian mucinase capable of hydrolyzing mucin. The mucinase is, among other things, suitable for counteracting diseases in which mucus is involved, such as cystic fibrosis, COPD, asthma, bronchitis, tuberculosis, tumors with altered mucus expression, and mucus-containing pathogens. The invention also provides a pharmaceutical composition comprising an effective amount of the mucinase and a method of therapeutic or prophylactic treatment of an individual against such a disease. Methods for obtaining the mucinase are also herewith provided, as well as nucleic acids encoding all or part of the mucinase. In one aspect, the invention provides a diagnostic kit comprising a mucinase, a mucinase-specific antibody, a mucinase-derived peptide, and/or nucleic acid encoding all or part of the mucinase.

Abstract: The invention provides a mammalian mucinase capable of hydrolyzing mucin. Said mucinase is among others suitable for counteracting diseases in which mucus is involved. Said diseases comprise cystic fibrosis, COPD, asthma, bronchitis, tuberculosis, tumours with altered mucus expression, and mucus-containing pathogens. The invention also provides a pharmaceutical composition comprising an effective amount of said mucinase and a method of therapeutic or prophylactic treatment of an individual against a disease in which mucus is involved. Methods for obtaining said mucinase are also herewith provided, as well as nucleic acids encoding (part of) said mucinase. In one aspect the invention provides a diagnostic kit comprising a mucinase, a mucinase-specific antibody, a mucinase-derived peptide and/or nucleic acid encoding (part of) said mucinase.