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

Comparative evaluation of liver-directed knockin strategies with viral and nonviral vectors in mouse inherited disease models.

在小鼠遗传疾病模型中,对以病毒载体和非病毒载体为靶点的肝脏基因敲入策略进行比较评价。

Jin Xiu, Wu Xiaoyi, Song Junwei, Luo Mei, Ye Qin, Ren Chengda, Song Li, Li Manjun, Hu Ming, An Yifang, Su Jing, Fu Jiamei, Xu Qiuxia, Luo Min, Liu Fanfei, Liu Man, Li Qiqi, Yao Shaohua, Chen Lu, Yang Yang

AAV-delivered engineered suppressor tRNA rescues visual function in mice with an inherited retinal disease.

利用 AAV 递送的工程化抑制 tRNA 可挽救患有遗传性视网膜疾病的小鼠的视觉功能。

Ren Chengda, Song Li, Hu Ming, Jin Xiu, Liang Licong, Liu Fanfei, Su Jing, Fu Jiamei, Yang Yiliu, Liu Man, Xu Qiuxia, Wu Xiaoyi, Luo Min, Ye Qin, Li Qiqi, An Yifang, Li Manjun, She Kaiqin, Wei Yuquan, Lu Fang, Yang Yang

Retinal Pigment Epithelium-Targeting Gene Therapy Corrects Ocular Symptoms in Mouse and Rat Models of Oculocutaneous Albinism Type I

视网膜色素上皮靶向基因疗法可纠正小鼠和大鼠I型眼皮肤白化病模型中的眼部症状

Song, Li; Ren, Chengda; Luo, Min; Su, Jing; Jin, Xiu; Fu, Jiamei; Xu, Qiuxia; Wu, Xiaoyi; Liu, Fanfei; Ye, Qin; Hu, Ming; Liu, Man; Li, Qiqi; An, Yifang; Li, Manjun; Wang, Qingnan; She, Kaiqin; Lu, Fang; Yang, Yang

Rescue of neurologic disease in mucopolysaccharidosis type II mice via AAV-mediated liver delivery of brain-penetrating iduronate-2-sulfatase.

通过 AAV 介导的肝脏递送脑穿透性艾杜糖醛酸-2-硫酸酯酶,挽救 II 型粘多糖贮积症小鼠的神经系统疾病。

Jin Xiu, Ye Qin, Wu Xiaoyi, Su Jing, Song Li, Fu Jiamei, Xu Qiuxia, Luo Min, Liu Fanfei, Ren Chengda, Hu Ming, Liu Man, An Yifang, Li Qiqi, Li Manjun, Yang Yang

RP1 Dual-AAV Gene Therapy Preserves Retinal Structure and Ameliorates Photoreceptor Degeneration in a Murine Model of Retinitis Pigmentosa.

RP1 双 AAV 基因疗法在视网膜色素变性小鼠模型中保护视网膜结构并改善感光细胞退化

Liu Fanfei, Li Qiqi, Su Jing, Fu Jiamei, Wu Xiaoyi, Xu Qiuxia, Ren Chengda, Jin Xiu, Song Li, Lu Fang, Yang Yang

Current developments of gene therapy in human diseases

人类疾病基因治疗的最新进展

Liu, Fanfei; Li, Ruiting; Zhu, Zilin; Yang, Yang; Lu, Fang

X-linked FRMD7 gene mutation in idiopathic congenital nystagmus and its role in eye movement: A case report and literature review

X连锁FRMD7基因突变与特发性先天性眼球震颤及其在眼球运动中的作用:病例报告及文献综述

Liu, Fanfei; Wang, Minjin; Liao, Meng; Liu, Longqian; Jiang, Xiaoshuang