	US 12,221,609 B2
	Compositions and methods to restore paternal UBE3A gene expression in human Angelman syndrome
Stormy Chamberlain, Oxford, CT (US); and Noelle Germain, Watertown, CT (US)
Assigned to University of Connecticut, Farmington, CT (US)
Appl. No. 17/271,982
Filed by University of Connecticut, Farmington, CT (US)
PCT Filed Sep. 20, 2019, PCT No. PCT/US2019/052272 § 371(c)(1), (2) Date Feb. 26, 2021, PCT Pub. No. WO2020/061528, PCT Pub. Date Mar. 26, 2020.
Claims priority of provisional application 62/734,435, filed on Sep. 21, 2018.
Prior Publication US 2021/0332368 A1, Oct. 28, 2021
Int. Cl. C12N 15/113 (2010.01); C12N 15/86 (2006.01)

CPC C12N 15/1137 (2013.01) [C12N 15/86 (2013.01); C12N 2310/121 (2013.01); C12N 2310/122 (2013.01); C12N 2310/128 (2013.01); C12N 2310/141 (2013.01); C12N 2310/531 (2013.01); C12N 2740/15042 (2013.01); C12N 2740/15043 (2013.01); C12N 2750/14142 (2013.01); C12N 2750/14143 (2013.01)]

28 Claims

1. A polynucleotide comprising a nucleotide sequence encoding a short hairpin RNA (shRNA), wherein the shRNA is capable of inhibiting the silencing of paternal UBE3A gene, and wherein the nucleotide sequence encoding the shRNA consists of SEQ ID NO: 2.