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:
Mar. 26, 2024

Filed:

Dec. 17, 2018
Applicant:

City of Hope, Duarte, CA (US);

Inventors:

Kevin V. Morris, Duarte, CA (US);

Tristan Scott, Duarte, CA (US);

Assignee:

City of Hope, Duarte, CA (US);

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C12N 9/22 (2006.01); C12N 15/11 (2006.01); C12N 15/115 (2010.01); C12N 15/90 (2006.01); A61K 38/00 (2006.01);
U.S. Cl.
CPC ...
C12N 15/11 (2013.01); C12N 9/22 (2013.01); C12N 15/115 (2013.01); C12N 15/907 (2013.01); A61K 38/00 (2013.01); C12N 2310/16 (2013.01); C12N 2310/20 (2017.05); C12N 2310/321 (2013.01); C12N 2310/322 (2013.01); C12N 2310/3519 (2013.01); C12N 2800/80 (2013.01);
Abstract

Provided are compositions and methods for altering gene expression in cells. The compositions and methods may utilize a nucleic acid sequence that has a genetically modified trans-activating crRNA (tracrRNA) sequence, where at least one uracil nucleotide of the tracrRNA sequence is replaced with a nucleotide other than uracil, and/or a nucleic acid sequence that has a guide RNA (gRNA) sequence wherein one or more cytosine nucleotides and/or one or more uracil nucleotides of said gRNA sequence are modified nucleotides. Also provided are methods of treating a disorder in a subject in need of the treatment. The method may involve administering to the subject the nucleic acid or a vector thereof in combination with an RNA-guided DNA endonuclease enzyme.


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