日期 影响因子
日期:
2020 年 — 2026 年
2020
2021
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2026
影响因子:
–

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

期刊:Science Translational Medicine
影响因子:14.6
doi:10.1126/scitranslmed.aam6375
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
微生物学
发表日期：2017
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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

期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1038/mtm.2016.49
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
发表日期：2016
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Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models.

基因疗法治疗威斯科特-奥尔德里奇综合征的临床前模型长期疗效和安全性证据

期刊:Molecular Therapy
影响因子:12
doi:10.1038/mt.2009.31
Marangoni Francesco, Bosticardo Marita, Charrier Sabine, Draghici Elena, Locci Michela, Scaramuzza Samantha, Panaroni Cristina, Ponzoni Maurilio, Sanvito Francesca, Doglioni Claudio, Liabeuf Marie, Gjata Bernard, Montus Marie, Siminovitch Katherine, Aiuti Alessandro, Naldini Luigi, Dupré Loïc, Roncarolo Maria Grazia, Galy Anne, Villa Anna
发表日期：2009
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