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:
Mar. 19, 2024

Filed:

Apr. 25, 2018
Applicant:

The Regents of the University of Colorado, a Body Corporate, Denver, CO (US);

Inventors:

Brian Freed, Aurora, CO (US);

Kirsten Anderson, Denver, CO (US);

Christina Roark, Englewood, CO (US);

Jennifer Matsuda, Denver, CO (US);

Attorney:
Primary Examiner:
Assistant Examiner:
Int. Cl.
CPC ...
C12N 15/85 (2006.01); A61K 35/28 (2015.01); A61K 48/00 (2006.01); C12N 5/0789 (2010.01); C12N 9/22 (2006.01); C12N 15/11 (2006.01);
U.S. Cl.
CPC ...
C12N 15/85 (2013.01); A01K 67/0275 (2013.01); A61K 35/28 (2013.01); C12N 5/0647 (2013.01); C12N 9/22 (2013.01); C12N 15/11 (2013.01); A01K 2217/05 (2013.01); A01K 2227/105 (2013.01); A01K 2267/0325 (2013.01); A61K 48/00 (2013.01); C12N 2310/20 (2017.05); C12N 2740/15042 (2013.01); C12N 2750/14142 (2013.01); C12N 2800/80 (2013.01);
Abstract

Methods of preventing or treating rheumatoid arthritis (RA) in a subject by introducing the DRB1*04:01mutation that is resistant to RA. The resistant allele is introduced into the subject having or at risk of developing RA, using a HLA CRISPR/Cas9 vector that targets codon 71 in the HLA allele DRB1*04:01, introducing a single A to G point mutation in codon 71 by homology directed repair to alter the lysine at position 71 of the expressed protein to glutamic acid. This modified allele is affected in the subject's hematopoietic stem cells, which are then expanded and transplanted back into the patient. This microgene therapy confers RA-resistance via an autologous transplant. The invention includes isolated nucleic acids, vectors, recombinant viruses, cells, and pharmaceutical compositions to modify the HLA DRB1*04:01 allele.


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