IP Library › Granted Patent US 12698513
Granted Patent B2
US 12698513 · App. 17/428,880 · Granted Aug 4, 2026

CRISPR compositions and methods for promoting gene editing of ribosomal protein S19 (RPS19) gene

Inventors: David Baram (Tel Aviv, IL); Lior Izhar (Tel Aviv, IL); Asael Herman (Ness-Ziona, IL); Rafi Emmanuel (Ramla, IL); Michal Golan Mashiach (Ness-Ziona, IL); Joseph Georgeson (Rehovot, IL)
Assignee: EmendoBio Inc.
C12N15/907A61K31/7105A61K35/28A61P7/06C12N5/0647C12N9/22C12N15/11C12N2310/20C12N2501/125C12N2501/22C12N2501/2303C12N2510/00C12N2800/80
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Quick Facts
Patent No.
US 12698513
App. No.
17/428,880
Granted
Aug 4, 2026
Kind
B2
Abstract

Isolated guide RNA (gRNA) molecules comprising CRISPR (crRNA) molecules that target the 19:41869823_T_C mutation of a Ribosomal Protein S19 (RPS19) allele.

Claims (10)

1 . An isolated guide RNA molecule (gRNA) that targets a mutant Ribosomal Protein S19 (RPS19) allele, wherein the gRNA comprises a CRISPR RNA (crRNA) molecule comprising the nucleic acid sequence set forth in SEQ ID NOs: 6269, 6357, 6449, 6315, 6405, or 6388.

2 . The isolated gRNA of claim 1 , wherein the crRNA molecule comprises the nucleic acid sequence set forth in SEQ ID NO: 6269.

3 . An isolated gRNA that targets a mutant RPS19 allele, wherein the gRNA comprises a crRNA molecule consisting of the nucleic acid sequence set forth in SEQ ID NO: 6269, 6357, 6449, 6315, 6405, or 6388.

4 . The isolated gRNA of claim 3 , wherein the crRNA molecule consists of the nucleic acid sequence set forth in SEQ ID NO: 6269.

5 . The isolated gRNA of claim 3 , wherein the crRNA molecule consists of the nucleic acid sequence set forth in SEQ ID NO: 6357.

6 . The isolated gRNA of claim 3 , wherein the crRNA molecule consists of the nucleic acid sequence set forth in SEQ ID NO: 6449.

7 . The isolated gRNA of claim 3 , wherein the crRNA molecule consists of the nucleic acid sequence set forth in SEQ ID NO: 6315.

8 . The isolated gRNA of claim 3 , wherein the crRNA molecule consists of the nucleic acid sequence set forth in SEQ ID NO: 6405.

9 . The isolated gRNA of claim 3 , wherein the gRNA comprises a crRNA consisting of the nucleic acid sequence set forth in SEQ ID NO: 6388.

10 . A composition comprising the isolated guide RNA molecule of claim 1 , a donor DNA template for homology directed repair (HDR), alteration, or replacement of a target sequence of the RPS19 allele, one or more CRISPR nucleases or sequences encoding the one or more CRISPR nucleases, and one or more tracrRNA molecules or sequences encoding the one or more tracrRNA molecules.