Nature Communications (Nov 2021)
Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models
- Chady H. Hakim,
- Sandeep R. P. Kumar,
- Dennis O. Pérez-López,
- Nalinda B. Wasala,
- Dong Zhang,
- Yongping Yue,
- James Teixeira,
- Xiufang Pan,
- Keqing Zhang,
- Emily D. Million,
- Christopher E. Nelson,
- Samantha Metzger,
- Jin Han,
- Jacqueline A. Louderman,
- Florian Schmidt,
- Feng Feng,
- Dirk Grimm,
- Bruce F. Smith,
- Gang Yao,
- N. Nora Yang,
- Charles A. Gersbach,
- Shi-jie Chen,
- Roland W. Herzog,
- Dongsheng Duan
Affiliations
- Chady H. Hakim
- Department of Molecular Microbiology and Immunology, The University of Missouri
- Sandeep R. P. Kumar
- Department of Pediatrics, Indiana University
- Dennis O. Pérez-López
- Department of Molecular Microbiology and Immunology, The University of Missouri
- Nalinda B. Wasala
- Department of Molecular Microbiology and Immunology, The University of Missouri
- Dong Zhang
- Department of Physics, The University of Missouri
- Yongping Yue
- Department of Molecular Microbiology and Immunology, The University of Missouri
- James Teixeira
- Department of Molecular Microbiology and Immunology, The University of Missouri
- Xiufang Pan
- Department of Molecular Microbiology and Immunology, The University of Missouri
- Keqing Zhang
- Department of Molecular Microbiology and Immunology, The University of Missouri
- Emily D. Million
- Department of Molecular Microbiology and Immunology, The University of Missouri
- Christopher E. Nelson
- Department of Biomedical Engineering, Duke University
- Samantha Metzger
- Department of Molecular Microbiology and Immunology, The University of Missouri
- Jin Han
- Department of Molecular Microbiology and Immunology, The University of Missouri
- Jacqueline A. Louderman
- Department of Molecular Microbiology and Immunology, The University of Missouri
- Florian Schmidt
- Department of Infectious Diseases/Virology, University of Heidelberg
- Feng Feng
- Department of Molecular Microbiology and Immunology, The University of Missouri
- Dirk Grimm
- Department of Infectious Diseases/Virology, University of Heidelberg
- Bruce F. Smith
- Department of Pathobiology, Auburn University
- Gang Yao
- Department of Biomedical, Biological & Chemical Engineering, The University of Missouri
- N. Nora Yang
- National Center for Advancing Translational Sciences, NIH
- Charles A. Gersbach
- Department of Biomedical Engineering, Duke University
- Shi-jie Chen
- Department of Physics, The University of Missouri
- Roland W. Herzog
- Department of Pediatrics, Indiana University
- Dongsheng Duan
- Department of Molecular Microbiology and Immunology, The University of Missouri
- DOI
- https://doi.org/10.1038/s41467-021-26830-7
- Journal volume & issue
-
Vol. 12,
no. 1
pp. 1 – 12
Abstract
The Cas9-specific T cell response has been speculated to impair CRISPR therapy. Here, the authors show that local and systemic AAV CRISPR therapy induces cytotoxic killing and eliminates rescued dystrophin in canine models of Duchenne muscular dystrophy.
