Precise CRISPR-Cas9 Gene Repair in Autologous Memory T Cells to Treat Familial Hemophagocytic Lymphohistiocytosis

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The authors used an adeno-associated virus–based CRISPR-Cas9 system with an inhibitor of nonhomologous end joining to repair such mutations in potentially long-lived T cells ex vivo.
[Science Immunology]
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