	US 12,258,566 B2
	Oligonucleotides for PRNP modulation
Anastasia Khvorova, Westborough, MA (US); and Zachary Kennedy, Worcester, MA (US)
Assigned to UNIVERSITY OF MASSACHUSETTS, Westborough, MA (US)
Filed by UNIVERSITY OF MASSACHUSETTS, Boston, MA (US)
Filed on Feb. 26, 2021, as Appl. No. 17/187,129.
Claims priority of provisional application 62/982,941, filed on Feb. 28, 2020.
Prior Publication US 2021/0317460 A1, Oct. 14, 2021
Int. Cl. C12N 15/113 (2010.01); C12Q 1/68 (2018.01)

CPC C12N 15/1138 (2013.01) [C12N 2310/14 (2013.01); C12N 2310/315 (2013.01); C12N 2310/3515 (2013.01); C12N 2320/30 (2013.01)]

50 Claims

1. An RNA molecule comprising:

about 8 nucleotides to 80 nucleotides in length; and

a double stranded RNA (dsRNA) comprising a sense strand and an antisense strand, each strand with a 5′ end and a 3′ end, wherein a portion of the antisense strand is complementary to a portion of the sense strand,

wherein the antisense strand comprises a sequence substantially complementary to a prion protein (PRNP) nucleic acid sequence of any one of SEQ ID Nos: 90, 159, 176, 179, and 181-183 to direct target-specific silencing, and

wherein the antisense strand comprises at least 70% 2′-O-methyl modifications.