Optimized CLN1 genes and expression cassettes and their use
AssigneeThe University of North Carolina at Chapel Hill
InventorSteven Gray
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Abstract
This invention relates to polynucleotides comprising a nucleotide sequence encoding a PPT1 polypeptide or a fragment thereof, vectors (viral or non-viral vectors) comprising the same, and methods of using the same for delivery of the open reading frame to a cell or a subject and to treat infantile neuronal lipofuscinosis (infantile Batten disease). The polynucleotides comprise an optimized CLN1 open reading frame.
Claims19
A polynucleotide comprising a human CLN1 open reading frame, wherein said human CLN1 open reading frame comprises the nucleotide sequence of SEQ ID NO:
- 1 or its complement.
An AAV9 vector comprising, in 3′ to 5′ order:
- a first AAV2 inverted terminal repeat (ITR), a cytomegalovirus enhancer, a chicken beta actin promoter, a hybrid/modified MVM intron, a human CLN1 open reading frame comprising the sequence of SEQ ID NO:
- 1, a bovine growth hormone polyadenylation site, and a second AV2 ITR.
Dependencies are inferred from the claim text.