Ocular vectors and uses thereof
AssigneeAldevron, LLC
InventorsRicardo SALDAÑA-MEYER, Jin HUH, Jodi KENNEDY
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Abstract
Provided herein are constructs for improved expression of transgenes (e.g., ocular transgenes), nucleic acid vectors thereof, pharmaceutical compositions thereof, and methods of use thereof (e.g., methods of treatment). The expression constructs include regulatory elements such as promoters, enhancers, insulators, and intronic sequences. Expression constructs, nucleic acid vectors and pharmaceutical compositions thereof, and methods of use thereof, disclosed herein can provide effective, durable treatments for ocular diseases.
Claims33
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A nucleic acid vector comprising:
- (a) a promoter comprising a native ABCA4 promoter or functional variant thereof;
- (b) a cDNA sequence encoding ABCA4;
- and
- (c) a regulatory element.
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A nucleic acid vector comprising:
- (a) a promoter comprising any one of SEQ ID NOs:
- 3-5;
- and
- (b) a cDNA sequence encoding MYO7A.
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Dependencies are inferred from the claim text.