Gene therapies for lysosomal disorders

A gene and vector technology, applied in the field of gene therapy for lysosomal disorders, can solve problems such as difficult treatment of Gaucher's disease
CN112481269APending Publication Date: 2021-03-12ПРЕВЕЙЛ ТЕРАПЬЮТИКС ИНК
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Patent Information

Authority / Receiving Office
CN · China
Patent Type
Applications(China)
Current Assignee / Owner
ПРЕВЕЙЛ ТЕРАПЬЮТИКС ИНК
Publication Date
2021-03-12

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Abstract

The present invention relates to gene therapies for lysosomal disorders. In particular, the present disclosure relates, in certain aspects, to compositions and methods for treating diseases associatedwith abnormal lysosomal function, such as Parkinson's disease (PD) and Gaucher's disease. In certain embodiments, the present disclosure provides expression constructs comprising a transfection geneencoding beta glucocerebrosidase (GBA), or a portion thereof, alone or in combination with one or more PD-associated genes. In certain embodiments, the present disclosure provides methods of treatingParkinson's disease by administering these expression constructs to a subject in need thereof.
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Description

[0001] The application date is October 3, 2018, the international application number PCT / US2018 / 054227 entered the Chinese national phase on May 22, 2020, the application number is 201880075846.8, and the title of the invention is "gene therapy for lysosomal disorders" divisional application of the application.

[0002] related application

[0003] This application claims U.S. Provisional Application Serial No. 62 / 567,296, filed October 3, 2017, entitled "GENETHERAPIES FOR LYSOSOMAL DISORDERS," 62 / 567,311 of "GENE THERAPIES FOR LYSOSOMALDISORDERS," filed October 3, 2017, "GENETHERAPIES FOR LYSOSOMAL DISORDERS" 62 / 567,319, 62 / 567,319, filed October 3, 2017, entitled "GENE THERAPIES FOR LYSOSOMAL DISORDERS," and 62 / 567,301, filed October 3, 2017, entitled "For 62 / 567,310 of GENE THERAPIES FOR LYSOSOMALDISORDERS for the benefit of 35 U.S.C. 119(e), the entire contents of each application are incorporated herein by reference. Background technique

[0004] Gaucher's disease is a...

Claims

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