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Precise CRISPR-Cas9 gene repair in autologous memory T cells to treat familial hemophagocytic lymphohistiocytosis.

Li X, Wirtz T, Weber T, Lebedin M, Lowenstein ED, Sommermann T, Zach A, Yasuda T, de la Rosa K, Chu VT, Schulte JH, Muller I, Kocks C, Rajewsky K
Sci Immunol. 2024 Feb 2;9(92):eadi0042. doi: 10.1126/sciimmunol.adi0042. Epub 2024 Feb 2. (Link opens in a new window) PubMed (Link opens in a new window) Article

Plasmids from Article

ID Plasmid Purpose
209073pDonor-Prf1-ires-mCherryTargeting vector for the mouse Prf1 locus to correct prf1 gene
209074pDonor-Prf1-mock-ires-mCherryTargeting vector for the mouse Prf1 locus to correct prf1 gene
209075pDonor-PRF1-Exon3Targeting vector for the human PRF1 locus to replace exon 3 with repaired exon 3
209076pDonor-PRF1-T2A-cDNATargeting vector for the human PRF1 locus to replace exon 3 with repaired cDNA

Antibodies from Article