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Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in mice.

Wang D, Li J, Song CQ, Tran K, Mou H, Wu PH, Tai PWL, Mendonca CA, Ren L, Wang BY, Su Q, Gessler DJ, Zamore PD, Xue W, Gao G
Nat Biotechnol. 2018 Oct;36(9):839-842. doi: 10.1038/nbt.4219. Epub 2018 Aug 13. (Link opens in a new window) PubMed (Link opens in a new window) Article

Plasmids from Article

ID Plasmid Purpose
121507pAAV.pU1a-SpCas9Expresses codon-optimized SpCas9 in mammalian cells.
121508pAAVsc.U6-sgFah.CMV/CB-EGFPExpresses sgRNA targeting mouse Fah intron 7 (sgFah).
121509pAAVsc.U6-sgFah.Intron9.CMV/CB-EGFPExpresses sgRNA targeting mouse Fah intron 9 (sgFah.Intron9).
121510pAAVsc.U6-sgAspa.CMV/CB-EGFPExpresses sgRNA targeting mouse Aspa intron 2 (sgAspa).
121511pAAVsc.U6-sgIdua.CMV/CB-EGFPExpresses sgRNA targeting mouse Idua intron 9 (sgIdua).
121512pAAVsc.U6-sgGTTG.CMV/CB-GlucExpresses sgRNA targeting the intron in the MADM alleles (GT and TG).

Antibodies from Article