Back to results
US 2013/0177549 A1Published application
Treatment of Synucleinopathies
AssigneeThe Brigham and Women's Hospital, Inc.
InventorsMichael Schlossmacher, Valerie Cullen, Lamya Shihabuddin, Seng H. Cheng
Life of this patent
Estimating the term…
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.
Claims14
A method of treating a subject with a synucleinopathy, but not a clinically diagnosed lysosomal storage disease, the method comprising administering to a subject any one or more of:
- an acid-beta-glucocerebrosidase (GBA) polypeptide,
- a polynucleotide encoding an acid-beta-glucocerebrosidase (GBA) polypeptide,
- a GBA polypeptide activating polypeptide,
- a polynucleotide encoding a GBA polypeptide activating agent,
- a cathepsin D polypeptide,
- a procathepsin D polypeptide, and
- a polynucleotide encoding a cathepsin D or procathepsin D polypeptide, in an amount effective to reduce a level of α-synuclein in the subject's central or peripheral nervous system, or both, or in the subject's lysosomal compartment.
4 more elements
Use of any one or more of
- an acid-beta-glucocerebrosidase (GBA) polypeptide,
- a polynucleotide encoding an acid-beta-glucocerebrosidase (GBA) polypeptide,
- a GBA polypeptide activating polypeptide,
- a polynucleotide encoding a GBA polypeptide activating agent,
- a cathepsin D polypeptide,
- a procathepsin D polypeptide, and
- a polynucleotide encoding a cathepsin D or procathepsin D polypeptide, in a method of preparing a medicament for the treatment of a synucleinopathy.
4 more elements
Dependencies are inferred from the claim text.