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

Enhanced lysosomal glycogen breakdown is associated with liver tumorigenesis in glycogen storage disease type III

溶酶体糖原分解增强与糖原贮积症III型患者的肝脏肿瘤发生有关。

Montalvo-Romeral, Valle; Jauze, Louisa; Perrot, Gwendoline; Amaouche, Mouna; Gardin, Antoine; Aguilar González, Araceli; Leblond, Alicia; Zitoun-Ardon, Carine; Evrard, Félicie; Cosette, Jérémie; Tatout, Christophe; Bordier, Fanny; Bertil-Froidevaux, Emilie; Georger, Christophe; van Wittenberghe, Laetitia; Paradis, Valérie; Gay, Simon; Dujardin, Fanny; Maillot, François; Gautier-Stein, Amandine; Mithieux, Gilles; Malfatti, Edoardo; Mussini, Charlotte; Labrune, Philippe; Mayeuf-Louchart, Alicia; Ronzitti, Giuseppe; Rajas, Fabienne

Identification of alterations of immunometabolism associated with Pompe disease

庞贝病相关免疫代谢改变的鉴定

Costa-Verdera, Helena; Gargaro, Marco; Cagin, Umut; Manni, Giorgia; Scalisi, Giulia; Veron, Philippe; Silva Barcelos, Estevão Carlos; Pieroni, Benedetta; Mencarelli, Giulia; Ricciuti, Doriana; Nemazanyy, Ivan; Sanatine, Peggy; van Wittenberghe, Laetitia; Laforêt, Pascal; Gross, David-Alexandre; Ronzitti, Giuseppe; Fallarino, Francesca; Mingozzi, Federico

AAV vectors trigger DNA damage response-dependent pro-inflammatory signalling in human iPSC-derived CNS models and mouse brain

AAV载体在人iPSC衍生的中枢神经系统模型和小鼠脑中触发DNA损伤反应依赖性促炎信号通路。

Costa-Verdera, Helena; Meneghini, Vasco; Fitzpatrick, Zachary; Abou Alezz, Monah; Fabyanic, Emily; Huang, Xin; Dzhashiashvili, Yulia; Ahiya, Avantika; Mangiameli, Elisabeth; Valeri, Erika; Crivicich, Giovanni; Piccolo, Silvia; Cuccovillo, Ivan; Caccia, Roberta; Chan, Ying Kai; Bertin, Bérangère; Ronzitti, Giuseppe; Engel, Esteban A; Merelli, Ivan; Mingozzi, Federico; Gritti, Angela; Kuranda, Klaudia; Kajaste-Rudnitski, Anna

SINEUP RNA rescues molecular phenotypes associated with CHD8 suppression in autism spectrum disorder model systems.

SINEUP RNA 可挽救自闭症谱系障碍模型系统中与 CHD8 抑制相关的分子表型

Di Leva Francesca, Arnoldi Michele, Santarelli Stefania, Massonot Mathieu, Lemée Marianne Victoria, Bon Carlotta, Pellegrini Miguel, Castellini Maria Elena, Zarantonello Giulia, Messina Andrea, Bozzi Yuri, Bernier Raphael, Zucchelli Silvia, Casarosa Simona, Dassi Erik, Ronzitti Giuseppe, Golzio Christelle, Morandell Jasmin, Gustincich Stefano, Espinoza Stefano, Biagioli Marta

Advancing AAV technology: From capsid design to scalable manufacturing

推进AAV技术:从衣壳设计到可扩展生产

Stone, Daniel; Mietzsch, Mario; Ronzitti, Giuseppe

Rescue of lysosomal acid lipase deficiency in mice by rAAV8 liver gene transfer.

利用 rAAV8 肝脏基因转移挽救小鼠溶酶体酸性脂肪酶缺乏症

Laurent Marine, Harb Rim, Jenny Christine, Oustelandt Julie, Jimenez Simon, Cosette Jeremie, Landini Francesca, Ferrante Aristide, Corre Guillaume, Vujic Nemanja, Piccoli Claudia, Brassier Anais, Van Wittenberghe Laetitia, Ronzitti Giuseppe, Kratky Dagmar, Pacelli Consiglia, Amendola Mario

Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I.

在克里格勒-纳贾尔综合征 I 型幼年大鼠和小鼠模型中重复进行 AAV 介导的肝脏基因治疗

Shi Xiaoxia, Bortolussi Giulia, Collaud Fanny, Le Brun Pierre-Romain, Bloemendaal Lysbeth Ten, Guerchet Nicolas, Rudi de Waart Dirk, Sellier Pauline, Duijst Suzanne, Veron Philippe, Mingozzi Federico, Kishimoto Takashi Kei, Ronzitti Giuseppe, Bosma Piter, Muro Andrés F

Overcoming the Challenges Imposed by Humoral Immunity to AAV Vectors to Achieve Safe and Efficient Gene Transfer in Seropositive Patients

克服体液免疫对AAV载体的挑战,实现血清阳性患者安全高效的基因转移

Gross, David-Alexandre; Tedesco, Novella; Leborgne, Christian; Ronzitti, Giuseppe

Human Immune Responses to Adeno-Associated Virus (AAV) Vectors

人体对腺相关病毒(AAV)载体的免疫反应

Ronzitti, Giuseppe; Gross, David-Alexandre; Mingozzi, Federico

Erratum: Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction

更正:低剂量肝脏靶向基因疗法通过诱导免疫耐受增强庞贝病酶替代疗法的疗效

Han, Sang-Oh; Ronzitti, Giuseppe; Arnson, Benjamin; Leborgne, Christian; Li, Songtao; Mingozzi, Federico; Koeberl, Dwight