US 12,270,057 B2
Optimized FIG4 genes and expression cassettes and their use
Steven James Gray, Southlake, TX (US); and Rachel Bailey, Trophy Club, TX (US)
Assigned to The University of North Carolina at Chapel Hill, Chapel Hill, NC (US)
Appl. No. 17/290,927
Filed by THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL, Chapel Hill, NC (US)
PCT Filed Nov. 5, 2019, PCT No. PCT/US2019/059752
§ 371(c)(1), (2) Date May 3, 2021,
PCT Pub. No. WO2020/097002, PCT Pub. Date May 14, 2020.
Claims priority of provisional application 62/755,871, filed on Nov. 5, 2018.
Prior Publication US 2021/0371837 A1, Dec. 2, 2021
Int. Cl. C12N 9/16 (2006.01); A61K 9/00 (2006.01); A61K 38/46 (2006.01); A61P 21/00 (2006.01); A61P 25/00 (2006.01); C12N 15/86 (2006.01); A61K 48/00 (2006.01)
CPC C12N 9/16 (2013.01) [A61K 9/0085 (2013.01); A61K 38/465 (2013.01); A61P 21/00 (2018.01); A61P 25/00 (2018.01); C12N 15/86 (2013.01); C12Y 301/03036 (2013.01); A61K 48/00 (2013.01); C12N 2750/14143 (2013.01); C12N 2800/22 (2013.01)] 23 Claims
 
1. A polynucleotide comprising a human FIG4 open reading frame, wherein the human FIG4 open reading frame is codon-optimized for expression in a human cell, wherein said human FIG4 open reading frame comprises the nucleotide sequence of SEQ ID NO: 1 or a nucleotide sequence having at least about 90% identity thereto.