	US 12,275,948 B2
	Optimized CLN5 genes and expression cassettes and their use
Steven James Gray, Southlake, TX (US); David Palmer, Christchurch (NZ); and Nadia Mitchell, Christchurch (NZ)
Assigned to The University of North Carolina at Chapel Hill, Chapel Hill, NC (US); and Lincoln University, Lincoln (NZ)
Appl. No. 17/254,725
Filed by THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL, Chapel Hill, NC (US); and LINCOLN UNIVERSITY, Lincoln (NZ)
PCT Filed Jun. 27, 2019, PCT No. PCT/US2019/039458 § 371(c)(1), (2) Date Dec. 21, 2020, PCT Pub. No. WO2020/006200, PCT Pub. Date Jan. 2, 2020.
Claims priority of provisional application 62/828,717, filed on Apr. 3, 2019.
Claims priority of provisional application 62/691,359, filed on Jun. 28, 2018.
Prior Publication US 2021/0269829 A1, Sep. 2, 2021
Int. Cl. C12N 15/85 (2006.01); A61P 25/28 (2006.01); C12N 15/86 (2006.01); C07H 21/04 (2006.01)

CPC C12N 15/86 (2013.01) [A61P 25/28 (2018.01); C07H 21/04 (2013.01); C12N 15/85 (2013.01); C12N 2750/14143 (2013.01); C12N 2830/008 (2013.01); C12N 2830/50 (2013.01)]

12 Claims

1. A polynucleotide comprising a human neuronal ceroid lipofuscinosis protein 5 (CLN5) open reading frame, wherein the human CLN5 open reading frame is codon-optimized for expression in a human cell, wherein the codon-optimized human open reading frame starts from the third start site of the wildtype human CLN5, and wherein the human CLN5 open reading frame comprises the nucleotide sequence of SEQ ID NO:1 or a nucleotide sequence having at least 90% identity thereto.