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.

Date of Patent:
Oct. 25, 2022

Filed:

May. 01, 2020
Applicant:

The Regents of the University of California, Oakland, CA (US);

Inventors:

Prashant Mali, La Jolla, CA (US);

Dhruva Katrekar, La Jolla, CA (US);

Assignee:
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/115 (2010.01); A61P 21/00 (2006.01); C12N 9/78 (2006.01); A61K 38/00 (2006.01); C12N 15/113 (2010.01);
U.S. Cl.
CPC ...
C12N 15/115 (2013.01); A61P 21/00 (2018.01); C12N 9/78 (2013.01); C12Y 305/04 (2013.01); A61K 38/00 (2013.01); C12N 15/113 (2013.01); C12N 15/1137 (2013.01); C12N 2310/20 (2017.05); C12N 2310/531 (2013.01); C12N 2320/34 (2013.01);
Abstract

Aspects of the disclosure relate to a gene therapy approach for diseases, disorders, or conditions caused by mutation in the stop codon utilizing modified tRNA. At least 10-15% of all genetic diseases, including muscular dystrophy (e.g. Duchene muscular dystrophy), some cancers, beta thalassemia, Hurler syndrome, and cystic fibrosis, fall into this category. Not to be bound by theory, it is believed that this approach is safer than CRISPR approaches due to minimal off-target effects and the lack of genome level changes.


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