http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-2021010032-A1

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filingDate 2020-09-17-04:00^^<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
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_05edd0138fe27e7588d650f5d0617561
publicationDate 2021-01-14-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber US-2021010032-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 and Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding beta-Glucocerebrosidase (GBA) or a portion thereof, Lysosomal Membrane Protein 2 (LIMP2), Prosaposin, or any combination of the foregoing. In some embodiments, the disclosure provides methods of Parkinson's disease by administering such expression constructs to a subject in need thereof
isCitedBy http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11661585-B2
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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