IP Library › Granted Patent US 12,472,239
Granted Patent B2
US 12,472,239 · App. 18/162,850 · Granted Nov 18, 2025

Guide RNA for repairing a mutant human titin gene using CRISPR technology

Inventors: Louise Rodino-Klapac (Grove City, OH); Rachael Potter (Dublin, OH)
Assignee: Research Institute at Nationwide Children's Hospital
A61K38/39A61K48/00A61K48/005A61P21/00C07K14/4716C12N9/22C12N15/11C12N15/113C12N15/88C12N15/907C12N2310/20C12N2750/14143C12N2800/80
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Quick Facts
Patent No.
US 12,472,239
App. No.
18/162,850
Granted
Nov 18, 2025
Kind
B2
Abstract

The present application provides materials and methods for treating a patient with a titin-based myopathy, particularly a titin-based cardiomyopathy, and/or other titinopathy. In addition, the present application provides materials and methods for editing the titin gene in a cell by genome editing.

Claims (14)

1 . An isolated nucleic acid that encodes a guide ribonucleic acid (gRNA) comprising a spacer sequence consisting of the nucleic acid sequence set forth in SEQ ID NO: 64, 66, 68, 70, 72, 74, or 76.

2 . The nucleic acid of claim 1 , wherein the gRNA is a single-molecule gRNA (sgRNA).

3 . The nucleic acid of claim 2 , wherein the gRNA is a modified gRNA or a modified single-molecule gRNA (sgRNA).

4 . A composition comprising the nucleic acid of claim 1 and an excipient.

5 . A vector comprising the nucleic acid of claim 1 .

6 . The vector of claim 4 , wherein the vector is a recombinant adeno-associated virus (AAV) vector.

7 . The AAV vector of claim 6 , wherein the AAV is AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10, AAV11, AAV12, AAV13, or AAVrh.74.

8 . A composition comprising the vector of claim 5 and an excipient.

9 . A nanoparticle, nanosphere, liposome, micelle, quantum dot, polyethylene glycol particle, or hydrogel comprising the nucleic acid of claim 1 .

10 . The nanoparticle of claim 9 , wherein the nanoparticle is a lipid nanoparticle.

11 . A composition comprising the nanoparticle, nanosphere, liposome, micelle, quantum dot, polyethylene glycol particle, or hydrogel of claim 9 and an excipient.

12 . A kit comprising the composition of claim 4 and instructions for use.

13 . A kit comprising the composition of claim 8 and instructions for use.

14 . A kit comprising the composition of claim 11 and instructions for use.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Aug 21, 2023
From: RODINO-KLAPAC, LOUISE, DR.; POTTER, RACHAEL
To: RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
Reel/Frame 064649/0270 →
Continuity (3)
Continuation 15776714
Provisional Application 62255887 · Nov 16, 2015
Related Publication 20230233651A1 · Jul 27, 2023
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