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.
Claims41
A polynucleotide comprising a human CLN1 open reading frame, wherein a polynucleotide sequence of the polynucleotide or its complement is codon-optimized for expression in a human cell.
An expression cassette comprising a polynucleotide comprising a human CLN1 open reading frame.
Dependencies are inferred from the claim text.