Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models
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.
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Nature Portfolio
2021
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oai:doaj.org-article:fa789cec57864e868cf64db8cae359c52021-11-28T12:31:07ZCas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models10.1038/s41467-021-26830-72041-1723https://doaj.org/article/fa789cec57864e868cf64db8cae359c52021-11-01T00:00:00Zhttps://doi.org/10.1038/s41467-021-26830-7https://doaj.org/toc/2041-1723The 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.Chady H. HakimSandeep R. P. KumarDennis O. Pérez-LópezNalinda B. WasalaDong ZhangYongping YueJames TeixeiraXiufang PanKeqing ZhangEmily D. MillionChristopher E. NelsonSamantha MetzgerJin HanJacqueline A. LoudermanFlorian SchmidtFeng FengDirk GrimmBruce F. SmithGang YaoN. Nora YangCharles A. GersbachShi-jie ChenRoland W. HerzogDongsheng DuanNature PortfolioarticleScienceQENNature Communications, Vol 12, Iss 1, Pp 1-12 (2021) |
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| topic |
Science Q |
| spellingShingle |
Science Q 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 Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models |
| description |
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. |
| format |
article |
| author |
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 |
| author_facet |
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 |
| author_sort |
Chady H. Hakim |
| title |
Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models |
| title_short |
Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models |
| title_full |
Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models |
| title_fullStr |
Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models |
| title_full_unstemmed |
Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models |
| title_sort |
cas9-specific immune responses compromise local and systemic aav crispr therapy in multiple dystrophic canine models |
| publisher |
Nature Portfolio |
| publishDate |
2021 |
| url |
https://doaj.org/article/fa789cec57864e868cf64db8cae359c5 |
| work_keys_str_mv |
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