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

A compact base editor rescues AATD-associated liver and lung disease in mouse models

一种紧凑型碱基编辑器可挽救小鼠模型中与 AATD 相关的肝脏和肺部疾病

Jenny Gao; Bamidele, Nathan; Pires-Ferreira, Debora; Zheng, Chunwei; Destefano, April; Cheng, Haoyang; Tang, Qiushi; Cao, Yueying; Xie, Jun; Gao, Guangping; Gruntman, Alisha; Sontheimer, Erik; Flotte, Terence R; Xue, Wen

A Compact Base Editor Rescues AATD-associated Liver and Lung Disease in Mouse Models.

紧凑型碱基编辑器可挽救小鼠模型中与 AATD 相关的肝脏和肺部疾病

Gao Jenny, Bamidele Nathan, Pires-Ferreira Debora, Destefano April, Tang Qiushi, Cao Yueying, Xie Jun, Gao Guangping, Gruntman Alisha, Sontheimer Erik, Flotte Terence R, Xue Wen

Limb Perfusion Delivery of a rAAV1 Alpha-1 Antitrypsin Vector in Non-Human Primates Is Safe but Insufficient for Therapy

在非人灵长类动物中,通过肢体灌注递送rAAV1 α-1抗胰蛋白酶载体是安全的,但不足以用于治疗。

Pires-Ferreira, Debora; Reil, Darcy; Tang, Qiushi; Blackwood, Meghan; Gallagher, Thomas; Keeler, Allison M; Chichester, Jessica A; Vyhnal, Kristin K; Lindborg, Jane A; Benson, Janet; Fu, Dongtao; Flotte, Terence R; Gruntman, Alisha M