Vemurafenib combined with cladribine and cytarabine results in durable remission of pediatric BRAF V600E–positive LCH

Autor: Evseev, Dmitry, Osipova, Daria, Kalinina, Irina, Raykina, Elena, Ignatova, Anna, Lyudovskikh, Evelina, Baidildina, Dina, Popov, Alexander, Zhogov, Vladimir, Semchenkova, Alexandra, Litvin, Eugeny, Kotskaya, Natalia, Cherniak, Ekaterina, Voronin, Kirill, Burtsev, Eugeny, Bronin, Gleb, Vlasova, Irina, Purbueva, Bazarma, Fink, Olesya, Pristanskova, Ekaterina, Dzhukaeva, Irina, Erega, Elena, Novichkova, Galina, Maschan, Alexey, Maschan, Michael

Publikováno v: In Blood Advances 26 September 2023 7(18):5246-5257

Gene-editing in patient and humanized-mice primary muscle stem cells rescues dysferlin expression in dysferlin-deficient muscular dystrophy.

Autor: Escobar H; Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany. helena.escobar@mdc-berlin.de.; Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Charité Campus Buch, Berlin, Germany. helena.escobar@mdc-berlin.de.; Muscle Research Unit, Experimental and Clinical Research Center (ECRC), a joint cooperation between the Charité-Universitätsmedizin Berlin and the Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany. helena.escobar@mdc-berlin.de., Di Francescantonio S; Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany.; Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Charité Campus Buch, Berlin, Germany.; Muscle Research Unit, Experimental and Clinical Research Center (ECRC), a joint cooperation between the Charité-Universitätsmedizin Berlin and the Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany., Smirnova J; Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany., Graf R; Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany.; Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Charité Campus Buch, Berlin, Germany.; Muscle Research Unit, Experimental and Clinical Research Center (ECRC), a joint cooperation between the Charité-Universitätsmedizin Berlin and the Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany., Müthel S; Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany.; Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Charité Campus Buch, Berlin, Germany.; Muscle Research Unit, Experimental and Clinical Research Center (ECRC), a joint cooperation between the Charité-Universitätsmedizin Berlin and the Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany., Marg A; Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany.; Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Charité Campus Buch, Berlin, Germany.; Muscle Research Unit, Experimental and Clinical Research Center (ECRC), a joint cooperation between the Charité-Universitätsmedizin Berlin and the Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany., Zhogov A; Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany.; Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Charité Campus Buch, Berlin, Germany.; Muscle Research Unit, Experimental and Clinical Research Center (ECRC), a joint cooperation between the Charité-Universitätsmedizin Berlin and the Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany., Krishna S; Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany.; Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Charité Campus Buch, Berlin, Germany.; Muscle Research Unit, Experimental and Clinical Research Center (ECRC), a joint cooperation between the Charité-Universitätsmedizin Berlin and the Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany.; Department of Biology, Chemistry, and Pharmacy, Freie Universität Berlin, Berlin, Germany., Metzler E; Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany.; Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Charité Campus Buch, Berlin, Germany.; Muscle Research Unit, Experimental and Clinical Research Center (ECRC), a joint cooperation between the Charité-Universitätsmedizin Berlin and the Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany., Petkova M; MyoPax GmbH, Berlin, Germany., Daumke O; Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany.; Department of Biology, Chemistry, and Pharmacy, Freie Universität Berlin, Berlin, Germany., Kühn R; Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany., Spuler S; Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany. simone.spuler@charite.de.; Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Charité Campus Buch, Berlin, Germany. simone.spuler@charite.de.; Muscle Research Unit, Experimental and Clinical Research Center (ECRC), a joint cooperation between the Charité-Universitätsmedizin Berlin and the Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany. simone.spuler@charite.de.; Department of Biology, Chemistry, and Pharmacy, Freie Universität Berlin, Berlin, Germany. simone.spuler@charite.de.

Publikováno v: Nature communications [Nat Commun] 2025 Jan 02; Vol. 16 (1), pp. 120. Date of Electronic Publication: 2025 Jan 02.

Vemurafenib combined with cladribine and cytarabine results in durable remission of pediatric BRAF V600E-positive LCH

Autor: Dmitry Evseev, Daria Osipova, Irina Kalinina, Elena Raykina, Anna Ignatova, Evelina Lyudovskikh, Dina Baidildina, Alexander Popov, Vladimir Zhogov, Alexandra Semchenkova, Eugeny Alexandrovich Litvin, Natalia Kotskaya, Ekaterina Cherniak, Kirill Voronin, Eugeny Burtsev, Gleb Bronin, Irina Vlasova, Bazarma Purbueva, Olesya Fink, Ekaterina Pristanskova, Irina Dzhukaeva, Elena Erega, Galina Novichkova, Alexey Maschan, Mikhail Maschan

Publikováno v: Blood Advances.

Langerhans cell histiocytosis (LCH) is a disorder with a variety of clinical signs. The most severe forms affect "risk organs" (RO). The established role of BRAF V600E mutation in LCH led to a targeted approach. However, the targeted therapy can't cu

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::65b7955f3d7b91c632c65c8466159cd9
https://doi.org/10.1182/bloodadvances.2022009067

Flow cell sorting followed by PCR ‐based clonality testing may assist in questionable diagnosis and monitoring of acute lymphoblastic leukemia

Autor: Alexandra Semchenkova, Vladimir Zhogov, Elena Zakharova, Ekaterina Mikhailova, Olga Illarionova, Sergey Larin, Galina Novichkova, Alexander Karachunskiy, Michael Maschan, Alexander Popov

Publikováno v: International Journal of Laboratory Hematology.

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::4c61ec29ecc402c9133e59542eeb0d46
https://doi.org/10.1111/ijlh.14053

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