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.
Claims22
A method of expressing a Ceroid-Lipofuscinosis, Neuronal 1 (CLN1) open reading frame in a cell, comprising contacting the cell with a polynucleotide comprising a human CLN1 open reading frame, wherein the human CLN1 open reading frame comprises the nucleotide sequence of SEQ ID NO:
- 1 or its complement, thereby expressing the CLNI open reading frame in the cell.
A method of expressing a CLN1 open reading frame in a subject, comprising delivering to the subject a polynucleotide comprising a human CLN1 open reading frame, wherein the human CLNI open reading frame comprises the nucleotide sequence of SEQ ID NO:
- 1 or its complement, thereby expressing the CLN1 open reading frame in the subject.
A method of treating a disorder associated with aberrant expression of a CLN1 gene or aberrant activity of a CLNI gene product in a subject in need thereof, comprising delivering to the subject a therapeutically effective amount of a polynucleotide comprising a human CLNI open reading frame, wherein the human CLNI open reading frame comprises the nucleotide sequence of SEQ ID NO:
- 1 or its complement, thereby treating the disorder associated with aberrant expression of the CLNI gene in the subject.
Dependencies are inferred from the claim text.