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Article

Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models

24 Nov 2021

Abstract excerpt

Adeno-associated virus (AAV)-mediated CRISPR-Cas9 editing holds promise to treat many diseases. The immune response to bacterial-derived Cas9 has been speculated as a hurdle for AAV-CRISPR therapy. However, immunological consequences of AAV-mediated Cas9 expression have thus far not been thoroughly investigated in large mammals. We evaluate Cas9-specific immune responses in canine models of Duchenne muscular...

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PMID: 34819506
DOI: 10.1038/s41467-021-26830-7

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