Compositions and methods for the treatment of lysosomal storage disorders and disorders characterized by lysosomal dysfunction
AssigneeBeyond Batten Disease Foundation
InventorMarco Sardiello
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Abstract
The present invention relates to compositions and methods of treating lysosomal storage diseases and methods of using trehalose.
Claims43
A method of treating a lysosomal storage disorder or disorder characterized by lysosomal dysfunction in a subject in need thereof, the method comprising administering a therapeutically effective amount of a composition comprising a protein kinase B inhibitor.
A method of using trehalose, the method comprising inhibiting the activity of a protein kinase B by contacting the protein kinase B with a composition comprising trehalose.
A method of enhancing clearance of undegraded material in a cell exhibiting dysfunctional lysosomal clearance, the method comprising inhibiting a protein kinase B in the cell by contacting the cell with a composition comprising a protein kinase B inhibitor.
Dependencies are inferred from the claim text.