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AssigneeVoyager Therapeutics, Inc.
InventorsHiu Yan CHUNG, Elisabeth KNOLL, Todd CARTER
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The disclosure relates to compositions and methods for altering, e.g., enhancing, the expression of GCase proteins, whether in vitro and/or in vivo. Such compositions include delivery of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Parkinson's Disease (PD), Gaucher Disease (GD), Dementia with Lewy Bodies (DLB), or related condition resulting from a deficiency in the quantity and/or function of GBA1 gene product or associated with decreased expression or protein levels of GCase protein.
An isolated nucleic acid comprising a nucleotide sequence that encodes a β-glucocerebrosidase 1 (GBA1) protein and is at least 93% identical (e.g., at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identical) to the nucleotide sequence of SEQ ID NO:
An isolated nucleic acid comprising a nucleotide sequence that encodes a β-glucocerebrosidase 1 (GBA1) protein and is at least 94% identical (e.g., at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, at least 99%, or 100% identical) to the nucleotide sequence of SEQ ID NO:
A recombinant viral genome comprising a nucleotide sequence encoding a β-glucocerebrosidase 1 (GBA1) protein, wherein the recombinant viral genome further comprises an miRNA (miR) binding site that modulates expression of the encoded GBA1 protein in a cell or tissue of the DRG, liver, hematopoietic lineage, or a combination thereof;
A recombinant viral genome comprising, in 5′ to 3′ order:
A recombinant viral genome comprising in 5′ to 3′ order:
A nucleic acid comprising the recombinant viral genome of any one of embodiments 8-34, and a backbone region suitable for replication of the viral genome in a cell, e.g., a bacterial cell (e.g., wherein the backbone region comprises one or both of a bacterial origin of replication and a selectable marker).
The method of 72 or 73, wherein the cell or tissue is a cell or tissue of the central nervous system (e.g., parenchyma).
Dependencies are inferred from the claim text.