USPTO Patent US12584149B2: Genome Editing of Primary Cells
Summary
The USPTO has granted patent US12584149B2 to The Board of Trustees of the Leland Stanford Junior University for methods of nuclease-mediated genome editing of primary cells. The patent covers techniques for inducing stable gene modification via homologous recombination and enriching genetically modified cells using DNA nucleases and AAV vectors.
What changed
USPTO Patent Grant US12584149B2, assigned to The Board of Trustees of the Leland Stanford Junior University, details methods for nuclease-mediated genome editing of primary cells. The patent focuses on inducing stable gene modification through homologous recombination and enriching genetically modified primary cells using DNA nucleases and adeno-associated viral (AAV) vectors. It also includes methods for preventing or treating diseases by administering these modified cells.
This patent grant represents a new intellectual property asset in the field of gene editing. While not a regulatory rule, it may impact future research and development activities for entities involved in biotechnology, gene therapy, and cell-based treatments. Companies operating in this space should be aware of this patent's claims, particularly concerning the use of Cas polypeptides and AAV vectors for homologous recombination in primary cells, to ensure freedom to operate and avoid potential infringement.
Source document (simplified)
Nuclease-mediated genome editing of primary cells and related kits
Grant US12584149B2 Kind: B2 Mar 24, 2026
Assignee
The Board of Trustees of the Leland Stanford Junior University
Inventors
Daniel P. Dever, Rasmus O. Bak, Ayal Hendel, Waracharee Srifa, Matthew H. Porteus
Abstract
In certain aspects, the present invention provides methods for inducing a stable gene modification of a target nucleic acid via homologous recombination in a primary cell, such as a primary blood cell and/or a primary mesenchymal cell. In certain other aspects, the present invention provides methods for enriching a population of genetically modified primary cells having targeted integration at a target nucleic acid. The methods of the present invention rely on the introduction of a DNA nuclease such as a Cas polypeptide and a homologous donor adeno-associated viral (AAV) vector into the primary cell to mediate targeted integration of the target nucleic acid. Also provided herein are methods for preventing or treating a disease in a subject in need thereof by administering to the subject any of the genetically modified primary cells or pharmaceutical compositions described herein to prevent the disease or ameliorate one or more symptoms of the disease.
CPC Classifications
C12N 15/907 C12N 5/0647 A61K 31/7115 A61K 35/28
Filing Date
2022-12-05
Application No.
18074781
Claims
44
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