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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 SA, Dai Z, Schmidt F, Rovai A, Fakhiri J, Dhingra A, Yuan Q, Yang T, Balakrishnan A, Steinbrück L, Srivaratharajan S, Manns MP, Schambach A, Grimm D, Bohne J, Sharma AD, Büning H, Ott M. Krooss SA, et al. iScience. 2020 Jan 24;23(1):100764. doi: 10.1016/j.isci.2019.100764. Epub 2019 Dec 12. iScience. 2020. PMID: 31887661 Free PMC article.
Correction: Pathological mechanism and antisense oligonucleotide-mediated rescue of a non-coding variant suppressing factor 9 RNA biogenesis leading to hemophilia B.
Krooss S, Werwitzke S, Kopp J, Rovai A, Varnholt D, Wachs AS, Goyenvalle A, Aartsma-Rus A, Ott M, Tiede A, Langemeier J, Bohne J. Krooss S, et al. PLoS Genet. 2021 Jan 28;17(1):e1009345. doi: 10.1371/journal.pgen.1009345. eCollection 2021 Jan. PLoS Genet. 2021. PMID: 33507897 Free PMC article.
Homozygous frame shift variant in ATP7B exon 1 leads to bypass of nonsense-mediated mRNA decay and to a protein capable of copper export.
Stalke A, Pfister ED, Baumann U, Eilers M, Schäffer V, Illig T, Auber B, Schlegelberger B, Brackmann R, Prokisch H, Krooss S, Bohne J, Skawran B. Stalke A, et al. Among authors: krooss s. Eur J Hum Genet. 2019 Jun;27(6):879-887. doi: 10.1038/s41431-019-0345-1. Epub 2019 Feb 5. Eur J Hum Genet. 2019. PMID: 30723317 Free PMC article.