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

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

Coupling AAV-mediated promoterless gene targeting to SaCas9 nuclease to efficiently correct liver metabolic diseases

将AAV介导的无启动子基因靶向与SaCas9核酸酶偶联,可有效纠正肝脏代谢疾病

De Caneva, Alessia; Porro, Fabiola; Bortolussi, Giulia; Sola, Riccardo; Lisjak, Michela; Barzel, Adi; Giacca, Mauro; Kay, Mark A; Vlahoviček, Kristian; Zentilin, Lorena; Muro, Andrés F

Promoterless gene targeting without nucleases rescues lethality of a Crigler-Najjar syndrome mouse model.

无需核酸酶的无启动子基因靶向可挽救克里格勒-纳贾尔综合征小鼠模型的致死性

Porro Fabiola, Bortolussi Giulia, Barzel Adi, De Caneva Alessia, Iaconcig Alessandra, Vodret Simone, Zentilin Lorena, Kay Mark A, Muro Andrés F

Generation of Ugt1-deficient murine liver cell lines using TALEN technology

利用TALEN技术构建Ugt1缺陷型小鼠肝细胞系

Porro, Fabiola; Bockor, Luka; De Caneva, Alessia; Bortolussi, Giulia; Muro, Andrés F

Regulated splicing of the fibronectin EDA exon is essential for proper skin wound healing and normal lifespan

纤连蛋白EDA外显子的调控剪接对于皮肤伤口的正常愈合和正常寿命至关重要。

Muro, Andres F; Chauhan, Anil K; Gajovic, Srecko; Iaconcig, Alessandra; Porro, Fabiola; Stanta, Giorgio; Baralle, Francisco E