	US 12,465,655 B2
	UBE3A genes and expression cassettes and their use
Benjamin David Philpot, Durham, NC (US); Steven James Gray, Southlake, TX (US); Charles Shyng, Apex, NC (US); and Matthew Judson, Chapel Hill, NC (US)
Assigned to The University of North Carolina at Chapel Hill, Chapel Hill, NC (US)
Appl. No. 17/612,833
Filed by The University of North Carolina at Chapel Hill, Chapel Hill, NC (US)
PCT Filed May 22, 2020, PCT No. PCT/US2020/034171 § 371(c)(1), (2) Date Nov. 19, 2021, PCT Pub. No. WO2020/237130, PCT Pub. Date Nov. 26, 2020.
Claims priority of provisional application 62/851,411, filed on May 22, 2019.
Prior Publication US 2022/0241434 A1, Aug. 4, 2022
Int. Cl. A61P 43/00 (2006.01); A61K 48/00 (2006.01); A61P 25/00 (2006.01); C12N 9/00 (2006.01); C12N 15/86 (2006.01)

CPC A61K 48/0058 (2013.01) [A61P 25/00 (2018.01); A61P 43/00 (2018.01); C12N 9/93 (2013.01); C12N 15/86 (2013.01); C12N 2800/22 (2013.01)]

16 Claims

1. A polynucleotide comprising a human UBE3A open reading frame, wherein the human UBE3A open reading frame encodes human UBE3A short isoform and long isoform, wherein the human UBE3A long isoform is human UBE3A long isoform 2 or human UBE3A long isoform 3,

wherein the polynucleotide comprises a first Kozak sequence comprising SEQ ID NO:6 operably linked to coding sequences for the long isoform and a second Kozak sequence comprising SEQ ID NO:8 operably linked to coding sequences for the short isoform; and

wherein the polynucleotide is devoid of a sequence encoding an exogenously added secretory signal.