Gene therapies for lysosomal disorders
AssigneePrevail Therapeutics, Inc.
InventorsAsa ABELIOVICH, Laura HECKMAN, Herve RHINN
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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.
Claims8
1-109. (canceled)
An isolated cell comprising:
- (i) a first vector encoding one or more adeno-associated virus (AAV) rep proteins and/or one or more AAV cap proteins;
- and
- (ii) a second vector comprising a nucleic acid comprising:
- (a) an expression construct comprising a promoter operably linked to a transgene insert encoding a Progranulin (PGRN) protein, wherein the transgene insert comprises the nucleotide sequence of SEQ ID NO:68;
- and
- (b) two adeno-associated virus (AAV) inverted terminal repeat (ITR) sequences flanking the expression construct.
A plasmid comprising a nucleic acid comprising:
- (i) an expression construct comprising a promoter operably linked to a transgene insert encoding a Progranulin (PGRN) protein, wherein the transgene insert comprises the nucleotide sequence of SEQ ID NO:68;
- and
- (ii) two adeno-associated virus (AAV) inverted terminal repeat (ITR) sequences flanking the expression construct.
Dependencies are inferred from the claim text.