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.
Patent No.:
Date of Patent:
Nov. 07, 2023
Filed:
Mar. 15, 2018
Applicant:
The Regents of the University of California, Oakland, CA (US);
Inventors:
Stephanie Cherqui, La Jolla, CA (US);
Eric Adler, La Jolla, CA (US);
Sylvia Evans, La Jolla, CA (US);
Assignee:
The Regents of the University of California, Oakland, CA (US);
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
A61K 35/28 (2015.01); A61K 35/14 (2015.01); C07K 14/705 (2006.01); C12N 15/86 (2006.01); A61P 3/00 (2006.01); A61K 38/00 (2006.01); A61K 48/00 (2006.01);
U.S. Cl.
CPC ...
A61K 35/28 (2013.01); A61K 35/14 (2013.01); A61P 3/00 (2018.01); C07K 14/705 (2013.01); C12N 15/86 (2013.01); A61K 38/00 (2013.01); A61K 48/00 (2013.01); C12N 2740/15043 (2013.01);
Abstract
Provided herein are methods for treating a lysosomal transmembrane protein disease or disorder through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject in need of treatment. Also provided are vectors containing the nucleic acid molecule.