日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

GM1 Oligosaccharide Ameliorates Rett Syndrome Phenotypes In Vitro and In Vivo via Trk Receptor Activation

GM1 寡糖通过激活 Trk 受体在体内和体外改善 Rett 综合征表型

Maria Fazzari, Giulia Lunghi, Emma Veronica Carsana, Manuela Valsecchi, Eleonora Spiombi, Martina Breccia, Silvia Rosanna Casati, Silvia Pedretti, Nico Mitro, Laura Mauri, Maria Grazia Ciampa, Sandro Sonnino, Nicoletta Landsberger, Angelisa Frasca, Elena Chiricozzi

Therapeutic advantages of combined gene/cell therapy strategies in a murine model of GM2 gangliosidosis

基因/细胞联合治疗策略在 GM2 神经节苷脂沉积症小鼠模型中的治疗优势

Davide Sala, Francesca Ornaghi, Francesco Morena, Chiara Argentati, Manuela Valsecchi, Valeria Alberizzi, Roberta Di Guardo, Alessandra Bolino, Massimo Aureli, Sabata Martino, Angela Gritti

Novel bicistronic lentiviral vectors correct β-Hexosaminidase deficiency in neural and hematopoietic stem cells and progeny: implications for in vivo and ex vivo gene therapy of GM2 gangliosidosis

新型双顺反子慢病毒载体可纠正神经和造血干细胞及子代细胞中的 β-己糖胺酶缺乏症:对 GM2 神经节苷脂沉积症的体内和体外基因治疗的意义

Francesca Ornaghi, Davide Sala, Fabiana Tedeschi, Maria Chiara Maffia, Martina Bazzucchi, Francesco Morena, Manuela Valsecchi, Massimo Aureli, Sabata Martino, Angela Gritti

Gangliosides in the differentiation process of primary neurons: the specific role of GM1-oligosaccharide

神经节苷脂在原代神经元分化过程中的作用:GM1-寡糖的具体作用

Erika Di Biase, Giulia Lunghi, Maria Fazzari, Margherita Maggioni, Diego Yuri Pomè, Manuela Valsecchi, Maura Samarani, Pamela Fato, Maria Grazia Ciampa, Simona Prioni, Laura Mauri, Sandro Sonnino, Elena Chiricozzi

Parkinson's disease recovery by GM1 oligosaccharide treatment in the B4galnt1+/- mouse model

GM1 寡糖治疗 B4galnt1+/- 小鼠模型中的帕金森病康复情况

Elena Chiricozzi, Laura Mauri, Giulia Lunghi, Erika Di Biase, Maria Fazzari, Margherita Maggioni, Manuela Valsecchi, Simona Prioni, Nicoletta Loberto, Diego Yuri Pomè, Maria Grazia Ciampa, Pamela Fato, Gianluca Verlengia, Stefano Cattaneo, Robert Assini, Gusheng Wu, Samar Alselehdar, Robert W Ledeen

iPSC-derived neurons from GBA1-associated Parkinson's disease patients show autophagic defects and impaired calcium homeostasis

GBA1 相关帕金森病患者的 iPSC 衍生神经元表现出自噬缺陷和钙稳态受损

David C Schöndorf, Massimo Aureli, Fiona E McAllister, Christopher J Hindley, Florian Mayer, Benjamin Schmid, S Pablo Sardi, Manuela Valsecchi, Susanna Hoffmann, Lukas Kristoffer Schwarz, Ulrike Hedrich, Daniela Berg, Lamya S Shihabuddin, Jing Hu, Jan Pruszak, Steven P Gygi, Sandro Sonnino, Thomas G