	US 11,866,702 B2
	Nucleic acid molecules for pseudouridylation
Bart Klein, Leiden (NL); Janne Juha Turunen, Leiden (NL); Lenka Van Sint Fiet, Leiden (NL); Pedro Duarte Morais Fernandes Arantes Da Silva, Leiden (NL); Julien Auguste Germain Boudet, Leiden (NL); Yi-Tao Yu, Pittsford, NY (US); Hironori Adachi, Rochester, NY (US); and Meemanage De Zoysa, Rochester, NY (US)
Assigned to University of Rochester, Rochester, NY (US); and ProQR Therapeutics II B.V., Leiden (NL)
Appl. No. 17/041,148
Filed by University of Rochester, Rochester, NY (US); and ProQR Therapeutics II B.V., Leiden (NL)
PCT Filed Mar. 27, 2019, PCT No. PCT/US2019/024282 § 371(c)(1), (2) Date Sep. 24, 2020, PCT Pub. No. WO2019/191232, PCT Pub. Date Oct. 3, 2019.
Claims priority of provisional application 62/648,648, filed on Mar. 27, 2018.
Prior Publication US 2021/0010002 A1, Jan. 14, 2021
Int. Cl. C07H 21/04 (2006.01); C12N 15/113 (2010.01)

CPC C12N 15/113 (2013.01) [C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2310/335 (2013.01)]

24 Claims

1. A nucleic acid molecule comprising a guide region consisting of a single hairpin structure corresponding to one of the two hairpin structures of the wild type H/ACA snoRNA, wherein

(i) the nucleic acid molecule is capable of forming a partially double stranded nucleic acid complex with a target RNA in a mammalian cell, wherein the target RNA comprises a target uridine;

(ii) the partially double stranded nucleic acid complex is capable of engaging a mammalian pseudouridylation enzyme;

(iii) the guide region assists in positioning the target uridine in the partially double stranded nucleic acid complex; and

(iv) the target uridine can be converted to a pseudouridine by the mammalian pseudouridylation enzyme.