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Number of items: 2.
2022
Riedmayr, Lisa M.; Hinrichsmeyer, Klara S.; Karguth, Nina; Böhm, Sybille; Splith, Victoria; Michalakis, Stylianos und Becirovic, Elvir
(2022):
dCas9-VPR-mediated transcriptional activation of functionally equivalent genes for gene therapy.
In: Nature Protocols, Vol. 17, No. 3: pp. 781-818
2020
Panagiotopoulos, Anna-Lena; Karguth, Nina; Pavlou, Marina; Boehm, Sybille; Gasparoni, Gilles; Walter, Joern; Graf, Alexander; Blum, Helmut; Biel, Martin; Riedmayr, Lisa Maria und Becirovic, Elvir
(2020):
Antisense Oligonucleotide- and CRISPR-Cas9-Mediated Rescue of mRNA Splicing for a Deep Intronic CLRN1 Mutation.
In: Molecular Therapy-Nucleic Acids, Vol. 21: pp. 1050-1061