In vivo adenine base editing reverts C282Y and improves iron metabolism in hemochromatosis mice
Rovai, Alice, Chung, BoMee, Hu, Qingluan, Hook, Sebastian, Yuan, Qinggong, Kempf, Tibor, Schmidt, Florian, Grimm, Dirk, Talbot, Steven R, Steinbrück, Lars, Götting, Jasper, Bohne, Jens, Krooss, Simon A, Ott, Michael
Published in Nature communications (05.09.2022)
Published in Nature communications (05.09.2022)
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Correction: Pathological mechanism and antisense oligonucleotide-mediated rescue of a non-coding variant suppressing factor 9 RNA biogenesis leading to hemophilia B
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Published in PLoS genetics (28.01.2021)
Published in PLoS genetics (28.01.2021)
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Pathological mechanism and antisense oligonucleotide-mediated rescue of a non-coding variant suppressing factor 9 RNA biogenesis leading to hemophilia B
Krooss, Simon, Werwitzke, Sonja, Kopp, Johannes, Rovai, Alice, Varnholt, Dirk, Wachs, Amelie S, Goyenvalle, Aurelie, Aarstma-Rus, Annemieke, Ott, Michael, Tiede, Andreas, Langemeier, Jörg, Bohne, Jens
Published in PLoS genetics (01.04.2020)
Published in PLoS genetics (01.04.2020)
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Ex Vivo/In vivo Gene Editing in Hepatocytes Using “All-in-One” CRISPR-Adeno-Associated Virus Vectors with a Self-Linearizing Repair Template
Krooss, Simon Alexander, Dai, Zhen, Schmidt, Florian, Rovai, Alice, Fakhiri, Julia, Dhingra, Akshay, Yuan, Qinggong, Yang, Taihua, Balakrishnan, Asha, Steinbrück, Lars, Srivaratharajan, Sangar, Manns, Michael Peter, Schambach, Axel, Grimm, Dirk, Bohne, Jens, Sharma, Amar Deep, Büning, Hildegard, Ott, Michael
Published in iScience (24.01.2020)
Published in iScience (24.01.2020)
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