Allosteric chaperones and uses thereof
AssigneesFondazione Telethon, Consiglio Nazionale Delle Ricerche
InventorsGiancarlo Parenti, Caterina Porto, Marco Moracci, Maria Carmina Ferrara, Beatrice Cobucci-Ponzano, Generoso Andria
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Abstract
The present invention relates to an allosteric non-inhibitory chaperone of the lysosomal acid alpha-glucosidase (GAA) for use in the treatment of a pathological condition characterized by a deficiency of the lysosomal acid alpha-glucosidase (GAA), to pharmaceutical composition thereof, to a method for increasing the activity of GAA in a subject and to a method for identifying an allosteric non-inhibitory chaperone for GAA.
Claims15
1-13. (canceled)
A method of treatment of a pathological condition characterized by a deficiency of the lysosomal acid alpha-glucosidase (GAA) comprising the administration of an effective dose of an allosteric non-inhibitory chaperone of the lysosomal acid alpha-glucosidase (GAA) to a patient in need thereof.
A method for increasing the activity of an endogenous and/or exogenous GAA in an individual suspected of suffering or suffering from a pathological condition characterized by a deficiency of the lysosomal acid alpha-glucosidase (GAA), which comprises administering to the individual an allosteric non-inhibitory chaperone of the lysosomal acid alpha-glucosidase (GAA) in an amount effective to increase activity of the endogenous and/or exogenous GAA in the individual.
26-27. (canceled)
A method for identifying an allosteric non-inhibitory chaperone for GAA comprising the steps of:
- a) labelling NAC and/or NAS and/or NAG chaperone with a fluorophore;
- b) adding to said labeled NAC and/or NAS and/or NAG an amount of rhGAA to obtain a basal rhGAA fluorescence;
- c) measuring the basal rhGAA fluorescence;
- d) adding a test agent;
- e) measuring the fluorescence of rhGAA;
- f) comparing the fluorescence of rhGAA measured in c) and e);
- wherein if a variation of intensity of fluorescence or if a variation of wavelength of fluorescence is observed then the test agent is an allosteric non-inhibitory chaperone for GAA.
Dependencies are inferred from the claim text.