Correction of a Factor VIII genomic inversion with designer-recombinases

Autor: Maciej Paszkowski-Rogacz, Frank Buchholz, Lukas Theo Schmitt, Takanori Takebe, Kentaro Iwasawa, Hoersten J, Masaki Kimura, Felix Lansing, Janet Karpinski, Rodriguez-Muela N, Grass T, Sonntag J, Günes C, Schneider Pm, Knöfler R, Rojo-Romanos T, Mukhametzyanova L

Despite advances in nuclease-based genome editing technologies, correcting human disease-causing genomic inversions remains a challenge. Here, we describe the potential use of a recombinase-based system to correct a 140 kb genomic inversion of the F8

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https://doi.org/10.1101/2020.11.02.328013

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Activation of recombinases at specific DNA loci by zinc-finger domain insertions.

Autor: Mukhametzyanova L; Medical Systems Biology, Medical Faculty, Technical University Dresden, Dresden, Germany., Schmitt LT; Medical Systems Biology, Medical Faculty, Technical University Dresden, Dresden, Germany.; Seamless Therapeutics GmbH, Dresden, Germany., Torres-Rivera J; Medical Systems Biology, Medical Faculty, Technical University Dresden, Dresden, Germany., Rojo-Romanos T; Medical Systems Biology, Medical Faculty, Technical University Dresden, Dresden, Germany.; Seamless Therapeutics GmbH, Dresden, Germany., Lansing F; Medical Systems Biology, Medical Faculty, Technical University Dresden, Dresden, Germany.; Seamless Therapeutics GmbH, Dresden, Germany., Paszkowski-Rogacz M; Medical Systems Biology, Medical Faculty, Technical University Dresden, Dresden, Germany., Hollak H; Medical Systems Biology, Medical Faculty, Technical University Dresden, Dresden, Germany.; Seamless Therapeutics GmbH, Dresden, Germany., Brux M; Medical Systems Biology, Medical Faculty, Technical University Dresden, Dresden, Germany., Augsburg M; Medical Systems Biology, Medical Faculty, Technical University Dresden, Dresden, Germany., Schneider PM; Medical Systems Biology, Medical Faculty, Technical University Dresden, Dresden, Germany.; Seamless Therapeutics GmbH, Dresden, Germany., Buchholz F; Medical Systems Biology, Medical Faculty, Technical University Dresden, Dresden, Germany. frank.buchholz@tu-dresden.de.

Publikováno v: Nature biotechnology [Nat Biotechnol] 2024 Dec; Vol. 42 (12), pp. 1844-1854. Date of Electronic Publication: 2024 Jan 31.