Mingozzi相关文献与解析，生知库 | 链接生命科学的每一步

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

代谢

免疫代谢

免疫/内分泌

发表日期：2026

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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。

期刊:Nature Communications

影响因子:15.7

doi:10.1038/s41467-025-58778-3

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

期刊:Molecular Therapy

影响因子:12

doi:10.1016/j.ymthe.2025.09.049

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

细胞生物学

发表日期：2025

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A myotropic AAV vector combined with skeletal muscle cis-regulatory elements improve glycogen clearance in mouse models of Pompe disease.

肌营养性 AAV 载体与骨骼肌顺式调控元件相结合，可改善庞贝氏症小鼠模型中的糖原清除

期刊:Molecular Therapy-Methods & Clinical Development

影响因子:4.7

doi:10.1016/j.omtm.2025.101464

Sellier P, Collaud F, Benchekroun Y Krimi, Jimenez V, Leon X, Daniele N, Pham Q H, El Andari J, VandenDriessche T, Chuah M K, Grimm D, Bosch F, Mingozzi F, Ronzitti G

Comparative Analysis of Tailored Minicraniotomy versus Standard Pterional Craniotomy in the Treatment of Anterior Circulation Aneurysms: A Single-Center Case-Control Observational Study

前循环动脉瘤治疗中，个体化小开颅术与标准翼点入路开颅术的比较分析：一项单中心病例对照观察性研究

期刊:Journal of Neurological Surgery Part B-Skull Base

影响因子:0.9

doi:10.1055/a-2399-0008

Dipellegrini, Giosuè; Boccaletti, Riccardo; Mingozzi, Anna; Sanna, Elisa; Policicchio, Domenico

Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy

肌肉定向 AAV 基因治疗治疗大鼠模型中的婴儿型庞贝氏症

期刊:Molecular Metabolism

影响因子:7

doi:10.1016/j.molmet.2024.101899

Sergio Muñoz, Joan Bertolin, Veronica Jimenez, Maria Luisa Jaén, Miquel Garcia, Anna Pujol, Laia Vilà, Victor Sacristan, Elena Barbon, Giuseppe Ronzitti, Jihad El Andari, Warut Tulalamba, Quang Hong Pham, Jesus Ruberte, Thierry VandenDriessche, Marinee K Chuah, Dirk Grimm, Federico Mingozzi, Fatima

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

期刊:Molecular Therapy-Methods & Clinical Development

影响因子:4.7

doi:10.1016/j.omtm.2024.101363

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 AndrÃ©s F

A Case of Acute Contact Dermatitis Caused by Lidocaine/Prilocaine Cream on a Zoon Balanitis

利多卡因/丙胺卡因乳膏引起Zoon龟头炎的急性接触性皮炎病例报告

期刊:Dermatology Practical & Conceptual

影响因子:2.3

doi:10.5826/dpc.1404a281

Robuffo, Silvia; Misciali, Cosimo; Lanzoni, Anna; Mingozzi, Anna; Piraccini, Bianca Maria

发表日期：2024

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Comparing Executive Functions in Children and Adolescents with Autism and ADHD-A Systematic Review and Meta-Analysis

比较自闭症和多动症儿童及青少年执行功能——系统评价和荟萃分析

期刊:Children-Basel

影响因子:2.1

doi:10.3390/children11040473

Ceruti, Claudia; Mingozzi, Alessandra; Scionti, Nicoletta; Marzocchi, Gian Marco

Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor

开发双杂交 AAV 载体用于内皮细胞靶向表达血管性血友病因子

期刊:Gene Therapy

影响因子:4.6

doi:10.1038/s41434-020-00218-6

Elena Barbon, Charlotte Kawecki, Solenne Marmier, Aboud Sakkal, Fanny Collaud, Severine Charles, Giuseppe Ronzitti, Caterina Casari, Olivier D Christophe, Cécile V Denis, Peter J Lenting, Federico Mingozzi

Identification of alterations of immunometabolism associated with Pompe disease

庞贝病相关免疫代谢改变的鉴定

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。

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

A myotropic AAV vector combined with skeletal muscle cis-regulatory elements improve glycogen clearance in mouse models of Pompe disease.

肌营养性 AAV 载体与骨骼肌顺式调控元件相结合，可改善庞贝氏症小鼠模型中的糖原清除

Comparative Analysis of Tailored Minicraniotomy versus Standard Pterional Craniotomy in the Treatment of Anterior Circulation Aneurysms: A Single-Center Case-Control Observational Study

前循环动脉瘤治疗中，个体化小开颅术与标准翼点入路开颅术的比较分析：一项单中心病例对照观察性研究

Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy

肌肉定向 AAV 基因治疗治疗大鼠模型中的婴儿型庞贝氏症

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

A Case of Acute Contact Dermatitis Caused by Lidocaine/Prilocaine Cream on a Zoon Balanitis

利多卡因/丙胺卡因乳膏引起Zoon龟头炎的急性接触性皮炎病例报告

Comparing Executive Functions in Children and Adolescents with Autism and ADHD-A Systematic Review and Meta-Analysis

比较自闭症和多动症儿童及青少年执行功能——系统评价和荟萃分析

Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor

开发双杂交 AAV 载体用于内皮细胞靶向表达血管性血友病因子