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Adenine Base Editing In Vivo with a Single Adeno-Associated Virus Vector.

Zhang H, Bamidele N, Liu P, Ojelabi O, Gao XD, Rodriguez T, Cheng H, Kelly K, Watts JK, Xie J, Gao G, Wolfe SA, Xue W, Sontheimer EJ
GEN Biotechnol. 2022 Jun 1;1(3):285-299. doi: 10.1089/genbio.2022.0015. Epub 2022 Jun 14. (Link opens in a new window) PubMed (Link opens in a new window) Article

Plasmids from Article

ID Plasmid Purpose
199261pEJS-HZ79 All-in-one AAV-U1a-NmeABE8e-2xBPSV40-U6-Rosa26_V2Optimized single AAV vector for expressing N-terminal fusion Nme2Cas9-ABE8e and one U6 driven sgRNA targeting mouse Rosa26 gene. This optimized construct showed improved in vivo editing efficiency.
199262pEJS1964 All-in-one AAV-U1a-NmeABE-8e-2xBPSV40-miniU6-Rosa26Single AAV vector for expressing N-terminal fusion Nme2Cas9-ABE8e and one miniU6 driven sgRNA targeting mouse Rosa26 gene
199263pEJS1830 All-in-one AAV-U1a-NmeABE-8e-2xBPSV40-U6-FahSingle AAV vector for expressing N-terminal fusion Nme2Cas9-ABE8e and one U6 driven sgRNA targeting the point mutation in the mouse Fah gene of a HT1 mouse model
199264pEJS1831 All-in-one AAV-U1a-NmeABE-8e-2xBPSV40-miniU6-FahSingle AAV vector for expressing N-terminal fusion Nme2Cas9-ABE8e and one miniU6 driven sgRNA targeting the point mutation in the mouse Fah gene of a HT1 mouse model
199265pEJS-HZ54 All-in-one AAV-U1a-NmeABE8e-2xBPSV40-U6-Rosa26Single AAV vector for expressing N-terminal fusion Nme2Cas9-ABE8e and one U6 driven sgRNA targeting mouse Rosa26 gene

Antibodies from Article