Pathological mechanism and antisense oligonucleotide-mediated rescue of a non-coding variant suppressing factor 9 RNA biogenesis leading to hemophilia B.

by Simon Krooss, Sonja Werwitzke, Johannes Kopp, Alice Rovai, Dirk Varnholt, Amelie S Wachs, Aurelie Goyenvalle, Annemieke Aarstma-Rus, Michael Ott, Andreas Tiede, Jörg Langemeier, Jens Bohne
Published 2020-04-01

Correction: Pathological mechanism and antisense oligonucleotide-mediated rescue of a non-coding variant suppressing factor 9 RNA biogenesis leading to hemophilia B.

by Simon Krooss, Sonja Werwitzke, Johannes Kopp, Alice Rovai, Dirk Varnholt, Amelie S Wachs, Aurelie Goyenvalle, Annemieke Aartsma-Rus, Michael Ott, Andreas Tiede, Jörg Langemeier, Jens Bohne
Published 2021-01-01

Ex Vivo/In vivo Gene Editing in Hepatocytes Using “All-in-One” CRISPR-Adeno-Associated Virus Vectors with a Self-Linearizing Repair Template

by Simon Alexander Krooss, Zhen Dai, Florian Schmidt, Alice Rovai, Julia Fakhiri, Akshay Dhingra, Qinggong Yuan, Taihua Yang, Asha Balakrishnan, Lars Steinbrück, Sangar Srivaratharajan, Michael Peter Manns, Axel Schambach, Dirk Grimm, Jens Bohne, Amar Deep Sharma, Hildegard Büning, Michael Ott
Published 2020-01-01