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

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

Overcoming barriers to commercially pre-viable gene and cell therapies for rare and ultra-rare diseases

克服罕见病和超罕见病基因和细胞疗法商业化前期可行性障碍

Barrett, David; Cannon, Paula M; Mingozzi, Federico; Porteus, Matthew; Rivière, Isabelle; Flotte, Terence R

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

Gene therapy access: Global challenges, opportunities, and views from Brazil, South Africa, and India

基因疗法普及:全球挑战、机遇以及来自巴西、南非和印度的观点

Cornetta, Kenneth; Bonamino, Martín; Mahlangu, Johnny; Mingozzi, Federico; Rangarajan, Savita; Rao, Jayandharan

Promoterless Gene Targeting Approach Combined to CRISPR/Cas9 Efficiently Corrects Hemophilia B Phenotype in Neonatal Mice

无启动子基因靶向方法结合 CRISPR/Cas9 技术可有效纠正新生小鼠的 B 型血友病表型

Lisjak, Michela; De Caneva, Alessia; Marais, Thibaut; Barbon, Elena; Biferi, Maria Grazia; Porro, Fabiola; Barzel, Adi; Zentilin, Lorena; Kay, Mark A; Mingozzi, Federico; Muro, Andrés F

Early Phase Clinical Immunogenicity of Valoctocogene Roxaparvovec, an AAV5-Mediated Gene Therapy for Hemophilia A

Valoctocogene Roxaparvovec(一种用于治疗血友病A的AAV5介导基因疗法)的早期临床免疫原性研究

Long, Brian R; Veron, Philippe; Kuranda, Klaudia; Hardet, Romain; Mitchell, Nina; Hayes, Gregory M; Wong, Wing Yen; Lau, Kelly; Li, Mingjin; Hock, M Benjamin; Zoog, Stephen J; Vettermann, Christian; Mingozzi, Federico; Schweighardt, Becky

Emerging Immunogenicity and Genotoxicity Considerations of Adeno-Associated Virus Vector Gene Therapy for Hemophilia

腺相关病毒载体基因疗法治疗血友病的免疫原性和基因毒性方面的新问题

Monahan, Paul E; Négrier, Claude; Tarantino, Michael; Valentino, Leonard A; Mingozzi, Federico

The Effect of CpG Sequences on Capsid-Specific CD8(+) T Cell Responses to AAV Vector Gene Transfer

CpG序列对AAV载体基因转移诱导的衣壳特异性CD8(+) T细胞反应的影响

Xiang, ZhiQuan; Kurupati, Raj K; Li, Yan; Kuranda, Klaudia; Zhou, Xiangyang; Mingozzi, Federico; High, Katherine A; Ertl, Hildegund C J

AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer

AAV载体在人体内的免疫原性:基因转移成功的漫长征程

Costa Verdera, Helena; Kuranda, Klaudia; Mingozzi, Federico