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US 2026/0209718 A1Published application
Gene therapies for lysosomal disorders
AssigneePrevail Therapeutics, Inc.
InventorsAsa ABELIOVICH, Laura HECKMAN, Herve RHINN, Li Chin WONG, Hsuan-Ni LIN, Franz HEFTI
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Abstract
The disclosure relates to compositions and methods for treatment of diseases associated with aberrant lysosomal function, such as fronto-temporal dementia (FTD). The disclosure also provides expression constructs comprising a transgene encoding progranulin or a portion thereof. The disclosure provides methods of treating FTD by administering such expression constructs to a subject in need thereof.
Claims9
1-16. (canceled)
A method of quantifying a progranulin (PGRN) protein level in a cerebrospinal fluid (CSF) sample, the method comprising:
- (1) diluting the CSF sample in a master mix containing dithiothreitol (DTT) and sample buffer;
- (2) loading the diluted CSF sample, an anti-progranulin antibody, a secondary antibody that detects the anti-progranulin antibody, luminol and peroxide into wells of a capillary cartridge;
- (3) loading the capillary cartridge into an automated Western blot immunoassay instrument;
- (4) using the automated Western blot immunoassay instrument to calculate signal intensity, peak area, and signal-to-noise ratio;
- and
- (5) quantifying a progranulin protein level in the CSF sample as the peak area of immunoreactivity to the anti-progranulin antibody.
3 more elements
Dependencies are inferred from the claim text.