Treatment of synucleinopathies
AssigneeThe Brigham and Women's Hospital, Inc.
InventorsMichael Schlossmacher, Valerie Cullen, Lamya Shihabuddin, Seng H. Cheng
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Abstract
This invention relates generally to treating synucleinopathies in subjects that are not clinically diagnosed with a lysosomal storage disease, as well as associated methods of making medicaments and screening methods.
Claims11
A method of treating a subject with a synucleinopathy, but not a clinically diagnosed lysosomal storage disease, the method comprising administering to a subject an acid-beta-glucocerebrosidase (GBA) polypeptide or a polynucleotide encoding an acid-beta-glucocerebrosidase (GBA) polypeptide in an amount effective to reduce a level of α-synuclein in the subject's nervous system or in the subject's lysosomal compartment.
Dependencies are inferred from the claim text.