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

Uncovering mitochondrial defects in photoreceptors opens therapeutic opportunities for Stargardt disease

发现光感受器中的线粒体缺陷为治疗斯塔加特病提供了契机。

Brillante, Simona; Volpe, Mariagrazia; Diana, Anna; Negueruela, Santiago; Molinari, Marta; Saurino, Rosa; Cipollaro, Eva; Polishchuk, Elena; Tenderini, Erika; Damiano, Carla; Tornabene, Patrizia; Polishchuk, Roman; Parenti, Giancarlo; Tarallo, Antonietta; Banfi, Sandro; Trapani, Ivana; Carrella, Sabrina; Indrieri, Alessia

Retinal gene therapy for Stargardt disease with dual AAV intein vectors is both safe and effective in large animal models.

使用双AAV内含子载体的视网膜基因疗法治疗Stargardt病在大动物模型中安全有效

Ferla Rita, Pugni Eugenio, Lupo Mariangela, Tiberi Paola, Fioretto Federica, Perota Andrea, Duchi Roberto, Lagutina Irina, Gesualdo Carlo, Rossi Settimio, Ventrella Domenico, Elmi Alberto, McClinton Benjamin, Toomes Carmel, Xu Tongzhou, Molday Robert S, Surace Enrico M, Simonelli Francesca, Bacci Maria L, Galli Cesare, Memon Muhammad A, Shams Naveed, Auricchio Alberto, Trapani Ivana

Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina

内含肽介导的蛋白质反式剪接扩大了视网膜中腺相关病毒的转移能力

Tornabene, Patrizia; Trapani, Ivana; Minopoli, Renato; Centrulo, Miriam; Lupo, Mariangela; de Simone, Sonia; Tiberi, Paola; Dell'Aquila, Fabio; Marrocco, Elena; Iodice, Carolina; Iuliano, Antonella; Gesualdo, Carlo; Rossi, Settimio; Giaquinto, Laura; Albert, Silvia; Hoyng, Carel B; Polishchuk, Elena; Cremers, Frans P M; Surace, Enrico M; Simonelli, Francesca; De Matteis, Maria A; Polishchuk, Roman; Auricchio, Alberto

Has retinal gene therapy come of age? From bench to bedside and back to bench

视网膜基因疗法是否已经成熟?从实验室到临床,再回到实验室

Trapani, Ivana; Auricchio, Alberto

Gene therapy of inherited retinal degenerations: prospects and challenges

遗传性视网膜变性疾病的基因治疗:前景与挑战

Trapani, Ivana; Banfi, Sandro; Simonelli, Francesca; Surace, Enrico M; Auricchio, Alberto

Vector platforms for gene therapy of inherited retinopathies

用于遗传性视网膜病基因治疗的载体平台

Trapani, Ivana; Puppo, Agostina; Auricchio, Alberto