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In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice.
Koblan LW, Erdos MR, Wilson C, Cabral WA, Levy JM, Xiong ZM, Tavarez UL, Davison LM, Gete YG, Mao X, Newby GA, Doherty SP, Narisu N, Sheng Q, Krilow C, Lin CY, Gordon LB, Cao K, Collins FS, Brown JD, Liu DR
Nature. 2021 Jan;589(7843):608-614. doi: 10.1038/s41586-020-03086-7. Epub 2021 Jan 6. PubMed Article

Plasmids from Article

ID Plasmid Purpose
154429hu6 HGPS sgRNA expression and ABE7.10max VRQR lentiviral plasmidhu6 HGPS sgRNA expression and ABE7.10max VRQR lentiviral plasmid
154430hu6 HGPS sgRNA expression and ABE7.10max VRQR C terminal AAV vectorhu6 HGPS sgRNA expression and ABE7.10max VRQR C terminal AAV vector

Antibodies from Article