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. 15, 2019

Filed:

Feb. 12, 2018
Applicant:

City of Hope, Duarte, CA (US);

Inventors:

Saswati Chatterjee, Altadena, CA (US);

Laura Jane Smith, Westford, MA (US);

Kamehameha Wong, Altadena, CA (US);

Assignee:

CITY OF HOPE, Duarte, CA (US);

Attorneys:
Primary Examiner:
Int. Cl.
CPC ...
A61K 48/00 (2006.01); A01N 63/00 (2006.01); C07H 19/00 (2006.01); C07H 21/00 (2006.01); C07K 14/005 (2006.01); C12N 15/90 (2006.01); C12N 15/86 (2006.01); C12N 7/00 (2006.01);
U.S. Cl.
CPC ...
C12N 15/907 (2013.01); A61K 48/005 (2013.01); A61K 48/0008 (2013.01); C07K 14/005 (2013.01); C12N 7/00 (2013.01); C12N 15/86 (2013.01); A61K 48/00 (2013.01); C12N 2710/16622 (2013.01); C12N 2750/14121 (2013.01); C12N 2750/14122 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14145 (2013.01); C12N 2750/14152 (2013.01); C12N 2750/14171 (2013.01); C12N 2800/70 (2013.01); C12N 2840/44 (2013.01);
Abstract

Adeno-associated virus (AAV) Clade F vectors or AAV vector variants (relative to AAV9) for precise editing of the genome of a cell and methods and kits thereof are provided. Targeted genome editing using the AAV Clade F vectors or AAV vector variants provided herein occurred at frequencies that were shown to be 1,000 to 100,000 fold more efficient than has previously been reported. Also provided are methods of treating a disease or disorder in a subject by editing the genome of a cell of the subject via transducing the cell with an AAV Clade F vector or AAV vector variant as described herein and further transplanting the transduced cell into the subject to treat the disease or disorder of the subject. Also provided herein are methods of treating a disease or disorder in a subject by in vivo genome editing by directly administering the AAV Clade F vector or AAV vector variant as described herein to the subject.


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