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

Intrabiliary infusion of naked DNA vectors targets periportal hepatocytes in mice

裸露 DNA 载体胆管内输注靶向小鼠门管周围肝细胞

Sereina Deplazes, Andrea Schlegel, Zhuolun Song, Gabriella Allegri, Nicole Rimann, Tanja Scherer, Melanie Willimann, Lennart Opitz, Sharon C Cunningham, Ian E Alexander, Anja Kipar, Johannes Häberle, Beat Thöny, Hiu Man Grisch-Chan

Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys

工程化肝嗜性 AAV 基因治疗食蟹猴鸟氨酸转氨甲酰酶缺乏症的安全性和有效性

Julien Baruteau, Sharon C Cunningham, Berna Seker Yilmaz, Dany P Perocheau, Simon Eaglestone, Derek Burke, Adrian J Thrasher, Simon N Waddington, Leszek Lisowski, Ian E Alexander, Paul Gissen

Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes

对 OTC 缺陷型患者来源的原代人肝细胞进行有效的体内编辑

Samantha L Ginn, Anais K Amaya, Sophia H Y Liao, Erhua Zhu, Sharon C Cunningham, Michael Lee, Claus V Hallwirth, Grant J Logan, Szun S Tay, Anthony J Cesare, Hilda A Pickett, Markus Grompe, Kimberley Dilworth, Leszek Lisowski, Ian E Alexander

Modeling correction of severe urea cycle defects in the growing murine liver using a hybrid recombinant adeno-associated virus/piggyBac transposase gene delivery system

使用混合重组腺相关病毒/piggyBac 转座酶基因递送系统对生长中的小鼠肝脏中严重的尿素循环缺陷进行建模校正

Sharon C Cunningham, Susan M Siew, Claus V Hallwirth, Christine Bolitho, Natsuki Sasaki, Gagan Garg, Iacovos P Michael, Nicola A Hetherington, Kevin Carpenter, Gustavo de Alencastro, Andras Nagy, Ian E Alexander

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

在异种移植肝模型中筛选和评估具有临床意义的AAV变体

Leszek Lisowski ,Allison P Dane ,Kirk Chu ,Yue Zhang ,Sharon C Cunningham ,Elizabeth M Wilson ,Sean Nygaard ,Markus Grompe ,Ian E Alexander ,Mark A Kay

Adeno-associated virus-mediated rescue of neonatal lethality in argininosuccinate synthetase-deficient mice

腺相关病毒介导挽救精氨琥珀酸合成酶缺陷小鼠的新生儿死亡

Cindy Y Kok, Sharon C Cunningham, Kevin H Carpenter, Allison P Dane, Susan M Siew, Grant J Logan, Philip W Kuchel, Ian E Alexander