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.
Claims15
A method for expressing PGRN protein in the central nervous system of a subject, the method comprising administering to the subject a rAAV comprising:
- (i) an AAV9 capsid protein;
- and
- (ii) a rAAV vector comprising a nucleic acid comprising an expression construct comprising a promoter operably linked to a transgene insert encoding a PGRN protein, wherein the transgene insert comprises the nucleotide sequence of SEQ ID NO:
- 68.
A method for expressing PGRN protein in the central nervous system of a subject, the method comprising administering to the subject a rAAV comprising:
- (i) an AAV9 capsid protein;
- and
- (ii) a rAAV vector comprising a nucleic acid comprising, in 5′ to 3′ order:
- (a) a first ITR;
- (b) a CMV enhancer;
- (c) a CBA promoter;
- (d) a transgene insert encoding a PGRN protein, wherein the transgene insert comprises the nucleotide sequence of SEQ ID NO:
- 68;
- (e) a WPRE;
- (f) a Bovine Growth Hormone polyA signal tail;
- and
- (g) a second ITR.
Dependencies are inferred from the claim text.