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:
Jan. 24, 2023

Filed:

Feb. 20, 2018
Applicant:

Crispr Therapeutics Ag, Zug, CH;

Inventors:

Ante Sven Lundberg, Cambridge, MA (US);

Samarth Kulkarni, Cambridge, MA (US);

Lawrence Klein, Cambridge, MA (US);

Hari Kumar Padmanabhan, Cambridge, MA (US);

Assignee:
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
A61K 48/00 (2006.01); A61K 38/46 (2006.01); C12N 15/10 (2006.01);
U.S. Cl.
CPC ...
A61K 48/005 (2013.01); A61K 38/465 (2013.01); C12N 15/102 (2013.01); C12N 2750/14143 (2013.01);
Abstract

The present disclosure provides materials and methods for treating a patient with one or more conditions or disorders associated with ATXN1 whether ex vivo or in vivo. For example, the present disclosure provides materials and methods for treating a patient with Spinocerebellar ataxia type 1 (SCA1). Also provided are materials and methods for editing a ATXN1 gene in a cell by genome editing. The present disclosure also provides materials and methods for altering the contiguous genomic sequence of a ATXN1 gene in a cell. In addition, the present disclosure provides one or more gRNAs for editing a ATXN1 gene. Also provided are therapeutics comprising at least one or more gRNAs for editing a ATXN1 gene. In addition, the present disclosure provides therapeutics for treating patients with a ATXN1 related condition or disorder.


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