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:
May. 09, 2023

Filed:

Apr. 19, 2018
Applicant:

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

Inventors:

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

Iram Khan, Issaquah, WA (US);

Assignee:

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

Attorney:
Primary Examiner:
Assistant Examiner:
Int. Cl.
CPC ...
C12N 15/90 (2006.01); C12N 9/22 (2006.01);
U.S. Cl.
CPC ...
C12N 15/907 (2013.01); C12N 9/22 (2013.01);
Abstract

Described herein are systems and methods for treating, inhibiting, or ameliorating X-linked disorders including Wiskott-Aldrich Syndrome (WAS) and X-linked thrombocytopenia (XLT) in subjects that have been identified or selected as being ones that would benefit from a therapy to treat, inhibit, or ameliorate WAS or XLT. The systems include nuclease and vector donor constructs configured for co-delivery to modify endogenous WAS locus.


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