The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Nov. 26, 2024
Filed:
Apr. 23, 2019
The Curators of the University of Missouri, Columbia, MO (US);
The Government of the United States of America As Represented BY the Secretary of the Department of Health and Human Services, National Institutes of Health, Rockville, MD (US);
Dongsheng Duan, Columbia, MO (US);
Chady Hakim, Columbia, MO (US);
Nalinda B. Wasala, Columbia, MO (US);
Yongping Yue, Columbia, MO (US);
The Curators of the University of Missouri, Columbia, MO (US);
The Government of the United States of America as Represented by the Secretary of the Department of Health and Human Services, Rockville, MD (US);
Abstract
Disclosed herein are methods for systemically editing a gene in a subject and for systemically treating a genetic condition in a subject using a dual-vector CRISPR-Cas therapy. The methods comprise administering to the subject, via systemic administration, a gene editing AAV vector encoding a CRISPR effector protein (e.g., a Cas protein) and a targeting AAV vector providing one or more gRNAs targeted to the gene. In the methods, the ratio of the targeting AAV vector to the gene editing vector is greater than or equal to 2. Also provided are dual-vector systems for editing a gene or treating a genetic disease in a subject.