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:
Aug. 01, 2023

Filed:

Apr. 25, 2019
Applicant:

Seattle Children's Hospital, Seattle, WA (US);

Inventors:

David J. Rawlings, Seattle, WA (US);

Iram F. Khan, Seattle, WA (US);

Yuchi Chiang Honaker, Seattle, WA (US);

Swati Singh, Seattle, WA (US);

Karen Sommer, Seattle, WA (US);

Andrew M. Scharenberg, Seattle, WA (US);

Assignee:

SEATTLE CHILDREN'S HOSPITAL, Seattle, WA (US);

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/87 (2006.01); C12N 15/00 (2006.01); C07H 21/04 (2006.01); A61K 48/00 (2006.01); C12N 15/113 (2010.01); C12N 9/22 (2006.01); A61K 35/15 (2015.01); C12N 5/0783 (2010.01); A01K 67/00 (2006.01);
U.S. Cl.
CPC ...
C12N 15/113 (2013.01); A61K 35/15 (2013.01); C12N 5/0637 (2013.01); C12N 9/22 (2013.01); C12N 2310/20 (2017.05); C12N 2501/60 (2013.01); C12N 2800/80 (2013.01);
Abstract

Aspects of the invention described herein concern the incorporation of a FOXP3 cDNA (e.g., full-length human codon-optimized cDNA) into a FOXP3 gene or a non-FOXP3 locus so as to provide constitutive or regulated FOXP3 expression in a primary human CD34cells or cells derived from edited CD34cells. In some embodiments, guide RNA sequences that are directed to FOXP3, AAVS1, or other candidate loci are used for CRISPR/Cas9-mediated gene regulation, and gene delivery cassettes for HDR based gene-modification are provided.


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