GM1-Ganglioside Accumulation at the Mitochondria-Associated ER Membranes Links ER Stress to Ca2+-Dependent Mitochondrial Apoptosis
Sano, Renata, Annunziata, Ida, Patterson, Annette, Moshiach, Simon, Gomero, Elida, Opferman, Joseph, Forte, Michael, d'Azzo, Alessandra
Published in Molecular cell (13.11.2009)
Published in Molecular cell (13.11.2009)
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Journal Article
Altered GM1 catabolism affects NMDAR-mediated Ca2+ signaling at ER-PM junctions and increases synaptic spine formation in a GM1-gangliosidosis model
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
Published in Cell reports (Cambridge) (28.05.2024)
Published in Cell reports (Cambridge) (28.05.2024)
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Journal Article
AAV-mediated gene therapy for galactosialidosis: A long-term safety and efficacy study
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
Published in Molecular therapy. Methods & clinical development (10.12.2021)
Published in Molecular therapy. Methods & clinical development (10.12.2021)
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Journal Article
Preclinical Enzyme Replacement Therapy with a Recombinant β-Galactosidase-Lectin Fusion for CNS Delivery and Treatment of GM1-Gangliosidosis
Weesner, Jason Andrew, Annunziata, Ida, Yang, Tianhong, Acosta, Walter, Gomero, Elida, Hu, Huimin, van de Vlekkert, Diantha, Ayala, Jorge, Qiu, Xiaohui, Fremuth, Leigh Ellen, Radin, David N, Cramer, Carole L, d'Azzo, Alessandra
Published in Cells (Basel, Switzerland) (01.08.2022)
Published in Cells (Basel, Switzerland) (01.08.2022)
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Journal Article
Galactosialidosis: preclinical enzyme replacement therapy in a mouse model of the disease, a proof of concept
Cadaoas, Jaclyn, Hu, Huimin, Boyle, Gabrielle, Gomero, Elida, Mosca, Rosario, Jayashankar, Kartika, Machado, Mike, Cullen, Sean, Guzman, Belle, van de Vlekkert, Diantha, Annunziata, Ida, Vellard, Michel, Kakkis, Emil, Koppaka, Vish, d’Azzo, Alessandra
Published in Molecular therapy. Methods & clinical development (12.03.2021)
Published in Molecular therapy. Methods & clinical development (12.03.2021)
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Journal Article
Muscle degeneration in neuraminidase 1-deficient mice results from infiltration of the muscle fibers by expanded connective tissue
Zanoteli, Edmar, de Vlekkert, Diantha van, Bonten, Erik J., Hu, Huimin, Mann, Linda, Gomero, Elida M., Harris, A. John, Ghersi, Giulio, d'Azzo, Alessandra
Published in Biochimica et biophysica acta (01.07.2010)
Published in Biochimica et biophysica acta (01.07.2010)
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Journal Article
GM1-ganglioside accumulation at the mitochondria-associated ER membranes links ER stress to Ca(2+)-dependent mitochondrial apoptosis
Sano, Renata, Annunziata, Ida, Patterson, Annette, Moshiach, Simon, Gomero, Elida, Opferman, Joseph, Forte, Michael, d'Azzo, Alessandra
Published in Molecular cell (13.11.2009)
Published in Molecular cell (13.11.2009)
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Journal Article
Lysosomal NEU1 deficiency affects amyloid precursor protein levels and amyloid-β secretion via deregulated lysosomal exocytosis
Annunziata, Ida, Patterson, Annette, Helton, Danielle, Hu, Huimin, Moshiach, Simon, Gomero, Elida, Nixon, Ralph, d’Azzo, Alessandra
Published in Nature communications (14.11.2013)
Published in Nature communications (14.11.2013)
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Journal Article
AAV-mediated gene therapy in a mouse model of sialidosis
van de Vlekkert, Diantha, Hu, Huimin, Weesner, Jason A., Gomero, Elida M., d'Azzo, Alessandra
Published in Molecular genetics and metabolism (01.02.2023)
Published in Molecular genetics and metabolism (01.02.2023)
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Journal Article
AAV-mediated gene therapy for sialidosis
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
Published in Molecular therapy (03.07.2024)
Published in Molecular therapy (03.07.2024)
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Journal Article
Preclinical enzyme replacement therapy with a recombinant β-galactosidase-lectin fusion for CNS delivery and treatment of GM1-gangliosidosis
Weesner, Jason A., van de Vlekkert, Diantha, Acosta, Walter L., Yang, Tianhong, Gomero, Elida M., Hu, Huimin, Radin, David, Cramer, Carole L., d'Azzo, Alessandra
Published in Molecular genetics and metabolism (01.02.2023)
Published in Molecular genetics and metabolism (01.02.2023)
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Journal Article
Chaperone mediated gene therapy for type I sialidosis
Annunziata, Ida, Hu, Huimin, Gomero, Elida, d'Azzo, Alessandra
Published in Molecular genetics and metabolism (01.02.2016)
Published in Molecular genetics and metabolism (01.02.2016)
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Journal Article
Altered GM1 catabolism affects NMDAR-mediated Ca 2+ signaling at ER-PM junctions and increases synaptic spine formation in a GM1-gangliosidosis model
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
Published in Cell reports (Cambridge) (28.05.2024)
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Published in Cell reports (Cambridge) (28.05.2024)
Journal Article
Preclinical Dose-Finding Study With a Liver-Tropic, Recombinant AAV-2/8 Vector in the Mouse Model of Galactosialidosis
Hu, Huimin, Gomero, Elida, Bonten, Erik, Gray, John T, Allay, Jim, Wu, Yanan, Wu, Jianrong, Calabrese, Christopher, Nienhuis, Arthur, d'Azzo, Alessandra
Published in Molecular therapy (01.02.2012)
Published in Molecular therapy (01.02.2012)
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Journal Article
Preclinical study of a scAAV2/8-Lp1-huPPCA vector in the treatment of the galactosialidosis mouse model
Hu, Huimin, Gomero, Elida, Bonten, Erik, Gray, John T, Nienhuis, Arthur, d'Azzo, Alessandra
Published in Molecular therapy (01.09.2013)
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Published in Molecular therapy (01.09.2013)
Journal Article
GM1-Ganglioside Accumulation at the Mitochondria-Associated ER Membranes Links ER Stress to Ca[super]2+-Dependent Mitochondrial Apoptosis
Sano, Renata, Annunziata, Ida, Patterson, Annette, Moshiach, Simon, Gomero, Elida, Opferman, Joseph, te, Michael, D'Azzo, Alessandra
Published in Molecular cell (13.11.2009)
Published in Molecular cell (13.11.2009)
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Journal Article
Lysosomal NEU1 deficiency affects amyloid precursor protein levels and amyloid-[beta] secretion via deregulated lysosomal exocytosis
Annunziata, Ida, Patterson, Annette, Helton, Danielle, Hu, Huimin, Moshiach, Simon, Gomero, Elida, Nixon, Ralph, D'azzo, Alessandra
Published in Nature communications (01.11.2013)
Published in Nature communications (01.11.2013)
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Journal Article