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γ-Secretase modulator resistance of an aggressive Alzheimer-causing presenilin mutant can be overcome in the heterozygous patient state by a set of advanced compounds by Johannes Trambauer, Rosa Maria Rodriguez Sarmiento, Holly J. Garringer, Katja Salbaum, Liliana D. Pedro, Dennis Crusius, Ruben Vidal, Bernardino Ghetti, Dominik Paquet, Karlheinz Baumann, Lothar Lindemann, Harald Steiner
Published 2025-02-01Get full text
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Correction: γ‑Secretase modulator resistance of an aggressive Alzheimer‑causing presenilin mutant can be overcome in the heterozygous patient state by a set of advanced compounds... by Johannes Trambauer, Rosa Maria Rodriguez Sarmiento, Holly J. Garringer, Katja Salbaum, Liliana D. Pedro, Dennis Crusius, Ruben Vidal, Bernardino Ghetti, Dominik Paquet, Karlheinz Baumann, Lothar Lindemann, Harald Steiner
Published 2025-04-01Get full text
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Repurposing of tamoxifen ameliorates CLN3 and CLN7 disease phenotype by Chiara Soldati, Irene Lopez‐Fabuel, Luca G Wanderlingh, Marina Garcia‐Macia, Jlenia Monfregola, Alessandra Esposito, Gennaro Napolitano, Marta Guevara‐Ferrer, Anna Scotto Rosato, Einar K Krogsaeter, Dominik Paquet, Christian M Grimm, Sandro Montefusco, Thomas Braulke, Stephan Storch, Sara E Mole, Maria A De Matteis, Andrea Ballabio, Julio L Sampaio, Tristan McKay, Ludger Johannes, Juan P Bolaños, Diego L Medina
Published 2021-08-01Get full text
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TPC2 rescues lysosomal storage in mucolipidosis type IV, Niemann–Pick type C1, and Batten disease by Anna Scotto Rosato, Einar K Krogsaeter, Dawid Jaślan, Carla Abrahamian, Sandro Montefusco, Chiara Soldati, Barbara Spix, Maria Teresa Pizzo, Giuseppina Grieco, Julia Böck, Amanda Wyatt, Daniela Wünkhaus, Marcel Passon, Marc Stieglitz, Marco Keller, Guido Hermey, Sandra Markmann, Doris Gruber‐Schoffnegger, Susan Cotman, Ludger Johannes, Dennis Crusius, Ulrich Boehm, Christian Wahl‐Schott, Martin Biel, Franz Bracher, Elvira De Leonibus, Elena Polishchuk, Diego L Medina, Dominik Paquet, Christian Grimm
Published 2022-08-01Get full text
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