US 12,257,322 B2
Optimized CLN1 genes and expression cassettes and their use
Steven Gray, Southlake, TX (US)
Assigned to The University of North Carolina at Chapel Hill, Chapel Hill, NC (US)
Filed by The University of North Carolina at Chapel Hill, Chapel Hill, NC (US)
Filed on Nov. 2, 2022, as Appl. No. 18/052,082.
Application 18/052,082 is a division of application No. 16/305,337, granted, now 11,504,435, previously published as PCT/US2017/037118, filed on Jun. 13, 2017.
Claims priority of provisional application 62/349,411, filed on Jun. 13, 2016.
Prior Publication US 2023/0372544 A1, Nov. 23, 2023
Int. Cl. A61K 48/00 (2006.01); C12N 15/86 (2006.01)
CPC A61K 48/0066 (2013.01) [C12N 15/86 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14145 (2013.01); C12N 2830/002 (2013.01)] 22 Claims
 
1. 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.