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

Synergism of dual AAV gene therapy and rapamycin rescues GSDIII phenotype in muscle and liver

双重AAV基因疗法与雷帕霉素的协同作用可挽救肌肉和肝脏中的GSDIII表型

Louisa Jauze ,Mallaury Vie ,Quentin Miagoux ,Lucille Rossiaud ,Patrice Vidal ,Valle Montalvo-Romeral ,Hanadi Saliba ,Margot Jarrige ,Helene Polveche ,Justine Nozi ,Pierre-Romain Le Brun ,Luca Bocchialini ,Amandine Francois ,Jérémie Cosette ,Jérémy Rouillon ,Fanny Collaud ,Fanny Bordier ,Emilie Bertil-Froidevaux ,Christophe Georger ,Laetitia van Wittenberghe ,Adeline Miranda ,Nathalie F Daniele ,David-Alexandre Gross ,Lucile Hoch ,Xavier Nissan ,Giuseppe Ronzitti

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

Editorial: Gene therapy and genome editing for metabolic liver disorders

社论:基因疗法和基因组编辑治疗代谢性肝病

Collaud, Fanny; Bortolussi, Giulia

Capsid-specific removal of circulating antibodies to adeno-associated virus vectors

衣壳特异性清除循环中针对腺相关病毒载体的抗体

Bertin, Berangere; Veron, Philippe; Leborgne, Christian; Deschamps, Jack-Yves; Moullec, Sophie; Fromes, Yves; Collaud, Fanny; Boutin, Sylvie; Latournerie, Virginie; van Wittenberghe, Laetitia; Delache, Benoit; Le Grand, Roger; Dereuddre-Bosquet, Nathalie; Benveniste, Olivier; Moullier, Philippe; Masurier, Carole; Merten, Otto; Mingozzi, Federico

Progress and challenges of gene therapy for Pompe disease

庞贝氏病基因治疗的进展与挑战

Ronzitti, Giuseppe; Collaud, Fanny; Laforet, Pascal; Mingozzi, Federico

Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase

利用腺相关病毒(AAV)介导的肝脏递送可分泌酸性α-葡萄糖苷酶挽救小鼠庞贝氏症

Puzzo, Francesco; Colella, Pasqualina; Biferi, Maria G; Bali, Deeksha; Paulk, Nicole K; Vidal, Patrice; Collaud, Fanny; Simon-Sola, Marcelo; Charles, Severine; Hardet, Romain; Leborgne, Christian; Meliani, Amine; Cohen-Tannoudji, Mathilde; Astord, Stephanie; Gjata, Bernard; Sellier, Pauline; van Wittenberghe, Laetitia; Vignaud, Alban; Boisgerault, Florence; Barkats, Martine; Laforet, Pascal; Kay, Mark A; Koeberl, Dwight D; Ronzitti, Giuseppe; Mingozzi, Federico

Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors

利用外泌体包裹的AAV载体增强肝脏基因转移并逃避预先存在的体液免疫

Meliani, Amine; Boisgerault, Florence; Fitzpatrick, Zachary; Marmier, Solenne; Leborgne, Christian; Collaud, Fanny; Simon Sola, Marcelo; Charles, Severine; Ronzitti, Giuseppe; Vignaud, Alban; van Wittenberghe, Laetitia; Marolleau, Beatrice; Jouen, Fabienne; Tan, Sisareuth; Boyer, Olivier; Christophe, Olivier; Brisson, Alain R; Maguire, Casey A; Mingozzi, Federico

Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics

利用转座子介导构建剪接突变的细胞和小鼠模型,以评估基于snRNA的疗法的疗效

Barbon, Elena; Ferrarese, Mattia; van Wittenberghe, Laetitia; Sanatine, Peggy; Ronzitti, Giuseppe; Collaud, Fanny; Colella, Pasqualina; Pinotti, Mirko; Mingozzi, Federico

A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome

经翻译优化的AAV-UGT1A1载体可安全、持久地矫正克里格勒-纳贾尔综合征

Ronzitti, Giuseppe; Bortolussi, Giulia; van Dijk, Remco; Collaud, Fanny; Charles, Severine; Leborgne, Christian; Vidal, Patrice; Martin, Samia; Gjata, Bernard; Sola, Marcelo Simon; van Wittenberghe, Laetitia; Vignaud, Alban; Veron, Philippe; Bosma, Piter J; Muro, Andres F; Mingozzi, Federico