Patent ID: 11946050
Assignee: APTAH BIO, INC.
Field: Biotechnology (Chemistry)
Classification: CPC C | IPC C

Claim 0:
1. An engineered polynucleotide comprising:
a) a first nucleotide sequence complementary to a splice site of a pre-messenger ribonucleic acid (pre-mRNA),
b) a recruiting nucleotide sequence comprising: (i) a nucleotide sequence complementary to at least 4 nucleotides of the Stem-Loop II (SL2) of an U1 snRNA, and/or (ii) a nucleotide sequence comprising a phosphorothioate internucleotide linkage that binds to a U1-C zinc finger, and
c) a second nucleotide sequence complementary to a sequence 5′ or 3′ to said splice site of the pre-mRNA, wherein said splice site and said sequence 5′ or 3′ to said splice site are separated by no more than five nucleotides;
wherein said recruiting nucleotide sequence comprises an apical loop, an upper stem adjacent to said apical loop, a lower stem, and an internal loop situated between said upper stem and said lower stem.