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filingDate 2020-04-06-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_98f2071eb7d10882ef53308ebccda81a
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_acfda7fcbd448d1ba7302e2a6a35c263
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publicationDate 2020-09-10-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber US-2020282080-A1
titleOfInvention Gene therapies for lysosomal disorders
abstract The disclosure relates, in some aspects, to compositions and methods for treatment of diseases associated with aberrant lysosomal function, for example Parkinson's disease (PD) and Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding beta-Glucocerebrosidase (GBA) or a portion thereof alone or in combination with one or more PD-associated genes. In some embodiments, the disclosure provides methods of Parkinson's disease by administering such expression constructs to a subject in need thereof.
priorityDate 2017-10-03-04:00^^<http://www.w3.org/2001/XMLSchema#date>
type http://data.epo.org/linked-data/def/patent/Publication

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