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

Claim 23:
24. An engineered polynucleotide comprising:
a) a first targeting moiety configured to specifically bind a pre-messenger ribonucleic acid (pre-mRNA) at a first targeted sequence therein, wherein the first targeting moiety comprises a sequence identical or complementary to 5′-GTCCA-3′,
b) a recruiting moiety comprising a sequence that is at least 90% similar or complementary to SEQ ID NO. 1 and is configured to recruit a spliceosomal moiety that comprises U1 snRNA and a U1-C protein, and
c) a second targeting moiety configured to specifically bind the pre-mRNA at a second targeted sequence therein, wherein the second targeting moiety comprises a sequence identical or complementary to 5′-CG-3′;
wherein said recruiting moiety 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.