CRISPR-Cas9 mediated proteinase 3 autoantigen deletion as a treatment strategy for anti-neutrophil cytoplasmic autoantibody-associated vasculitis
We show the feasibility and efficacy of depleting the PR3 autoantigen in human CD34<sup>+</sup> HSPCs using CRISPR-Cas9. Depleting the PR3 autoantigen instead of suppressing the autoimmune response to PR3 could potentially lead to drug-free remission, particularly in patients with refractory or relapsing disease.
Authors
- Dimitrios L. Wagner (ORCID: https://orcid.org/0000-0002-2189-3579)
- Claudia Eulenberg-Gustavus (ORCID: https://orcid.org/0000-0002-2425-2275)
- Ralph Kettritz (ORCID: https://orcid.org/0000-0001-5821-6718)
- Uwe Jerke (ORCID: https://orcid.org/0000-0001-6887-8010)
- Adrian Schreiber (ORCID: https://orcid.org/0000-0003-1244-6379)
Institutions
- Max Delbrück Center (DE)
- Humboldt-Universität zu Berlin (DE)
- Berlin Institute of Health at Charité - Universitätsmedizin Berlin (DE)
- Freie Universität Berlin (DE)
- Charité - Universitätsmedizin Berlin (DE)
Publication Details
- Journal
- Kidney International
- Published
- 2025-04-21
- DOI
- https://doi.org/10.1016/j.kint.2025.03.020
- Citations
- 1
- Primary Topic
- Vasculitis and related conditions
- Type
- article
- Field-Weighted Citation Impact
- 1.52
Funders
- Deutsche Forschungsgemeinschaft