Altered GM1 catabolism affects NMDAR-mediated Ca2+ signaling at ER-PM junctions and increases synaptic spine formation in a GM1-gangliosidosis model von Weesner, Jason A., Annunziata, Ida, van de Vlekkert, Diantha, Robinson, Camenzind G., Campos, Yvan, Mishra, Ashutosh, Fremuth, Leigh E., Gomero, Elida, Hu, Huimin, d’Azzo, Alessandra

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AAV-mediated gene therapy for galactosialidosis: A long-term safety and efficacy study von Hu, Huimin, Mosca, Rosario, Gomero, Elida, van de Vlekkert, Diantha, Campos, Yvan, Fremuth, Leigh E., Brown, Scott A., Weesner, Jason A., Annunziata, Ida, d’Azzo, Alessandra

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AAV-mediated gene therapy for sialidosis von van de Vlekkert, Diantha, Hu, Huimin, Weesner, Jason A., Fremuth, Leigh E., Brown, Scott A., Lu, Meifen, Gomero, Elida, Campos, Yvan, Sheppard, Heather, d’Azzo, Alessandra

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Conventional and Unconventional Therapeutic Strategies for Sialidosis Type I von Mosca, Rosario, van de Vlekkert, Diantha, Campos, Yvan, Fremuth, Leigh E., Cadaoas, Jaclyn, Koppaka, Vish, Kakkis, Emil, Tifft, Cynthia, Toro, Camilo, Allievi, Simona, Gellera, Cinzia, Canafoglia, Laura, Visser, Gepke, Annunziata, Ida, d'Azzo, Alessandra

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AAV-mediated gene therapy for galactosialidosis: A long-term safety and efficacy study von Fremuth, Leigh E., Hu, Huimin, van de Vlekkert, Diantha, Weesner, Jason A., Gomero, Elida M., d'Azzo, Alessandra

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From mouse to sheep: Generating a sheep model and developing a gene therapy for sialidosis von Gallagher, Jillian, Gallant, Judith, Xu, Ping, Gately, Rachael, Bertrand, Stephanie, McElroy, Abigail, Hall, Erin, Benatti, Hector, Prestigiacomo, Rachel, Luk, Kevin, Fremuth, Leigh E., van de Vlekkert, Diantha, Hu, Huimin, Toro, Camilo, d'Azzo, Alessandra, Tifft, Cynthia J., Wolfe, Scot, Rivera, Jaime, Sena-Esteves, Miguel, Gray-Edwards, Heather

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Altered GM1 catabolism affects NMDAR-mediated Ca 2+ signaling at ER-PM junctions and increases synaptic spine formation in a GM1-gangliosidosis model von Weesner, Jason A, Annunziata, Ida, van de Vlekkert, Diantha, Robinson, Camenzind G, Campos, Yvan, Mishra, Ashutosh, Fremuth, Leigh E, Gomero, Elida, Hu, Huimin, d'Azzo, Alessandra

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Conventional and Unconventional Therapeutic Strategies for Sialidosis Type I von Mosca, Rosario, van de Vlekkert, Diantha, Campos, Yvan, Fremuth, Leigh E, Cadaoas, Jaclyn, Koppaka, Vish, Kakkis, Emil, Tifft, Cynthia, Toro, Camilo, Allievi, Simona, Gellera, Cinzia, Canafoglia, Laura, Visser, Gepke, Annunziata, Ida, d'Azzo, Alessandra

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