Gene therapies for lysosomal disorders
AssigneePrevail Therapeutics, Inc.
InventorsAsa Abeliovich, Laura Heckman, Herve Rhinn, Li Chin Wong, Hsuan-Ni Lin, Franz Hefti
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Abstract
The disclosure relates to compositions and methods for treatment of diseases associated with aberrant lysosomal function, such as fronto-temporal dementia (FTD). The disclosure also provides expression constructs comprising a transgene encoding progranulin or a portion thereof. The disclosure provides methods of treating FTD by administering such expression constructs to a subject in need thereof.
Claims13
A method for treating a subject having fronto-temporal dementia with a GRN mutation, the method comprising administering to the subject a recombinant adeno-associated virus (rAAV) comprising:
- (i) a rAAV vector comprising a nucleic acid comprising 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) an AAV9 capsid protein;
- wherein the rAAV is administered via an injection into the cisterna magna.
A method for treating a subject having fronto-temporal dementia with a GRN mutation, the method comprising administering to the subject a rAAV comprising:
- (i) a rAAV vector comprising a nucleic acid comprising, in 5′ to 3′ order:
- (a) an AAV2 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) an AAV2 ITR;
- and
- (ii) an AAV9 capsid protein;
- wherein the rAAV is administered via an injection into the cisterna magna.
Dependencies are inferred from the claim text.