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

Targeted editing of the 21-hydroxylase locus confers durable therapeutic effect in a murine model of congenital adrenal hyperplasia

针对21-羟化酶基因座的靶向编辑在先天性肾上腺增生症小鼠模型中具有持久的治疗效果

Graves, Lara E; Viswanath, Lakshmy; van Dijk, Eva B; Zhu, Erhua; Koyyalamudi, Sundar; Wotton, Tiffany; Rosser, Sebastian; Srinivasan, Shubha; Ginn, Samantha L; Alexander, Ian E

Pancreatic transdifferentiation of NOD mouse livers prevented development of hyperglycemia.

NOD小鼠肝脏的胰腺转分化可阻止高血糖症的发生。

Mahoney Alexandra L G, Ren Binhai, Nassif Najah T, O'Brien Bronwyn A, Logan Grant J, Gorrie Catherine A, Alexander Ian E, Simpson Ann M

A novel fusion protein reduces kidney complement in experimental C3 glomerulopathy

一种新型融合蛋白可降低实验性C3肾小球病中的肾脏补体水平。

Malik, Talat H; Kwiatkowska, Karolina; Lomax-Browne, Hannah J; Bottomley, Charlotte M; Bright, Matthew; Tean, Zhang Sung; Akturk, Ahmet K; Alexander, Ian E; Logan, Grant J; Pickering, Matthew C

High-throughput evaluation of cardiac-specific promoters for adeno-associated virus mediated cardiac gene therapy

利用高通量方法评估腺相关病毒介导的心脏基因治疗中心脏特异性启动子

Ravindran, Dhanya; Rao, Renuka; Mundisugih, Juan; Titus, Tracy; Tsurusaki, Shinya; Kok, Cindy Y; Rashid, Fairooj N; Igoor, Sindhu; Kotake, Yasuhito; Kumar, Saurabh; Chong, James J H; Alexander, Ian E; Lisowski, Leszek; Kizana, Eddy

Structural characterization of antibody-responses following Zolgensma treatment for AAV capsid engineering to expand patient cohorts.

对Zolgensma治疗后抗体反应的结构进行表征,以进行AAV衣壳工程改造,从而扩大患者群体

Mietzsch Mario, Hsi Jane, Nelson Austin R, Khandekar Neeta, Huang Ann-Maree, Smith Nicholas Jc, Zachary Jon, Potts Lindsay, Farrar Michelle A, Chipman Paul, Ghanem Mohammad, Alexander Ian E, Logan Grant J, Huiskonen Juha T, McKenna Robert

Gene therapy enhances deoxyribonuclease I treatment in antimyeloperoxidase glomerulonephritis.

基因疗法增强了抗髓过氧化物酶肾小球肾炎中脱氧核糖核酸酶 I 的治疗效果

Cao Le Anne, Oudin Virginie, Dick Jonathan, Alikhan Maliha A, Gottschalk Timothy A, Lu Lu, Lawlor Kate E, Koo Yuk Cheong Daniel, Mandwie Mawj, Alexander Ian E, Kitching A R, Gan Poh-Yi, Logan Grant J, O'Sullivan Kim M

Exploration of the potential of genomic editing in the treatment of congenital adrenal hyperplasia

探索基因组编辑在治疗先天性肾上腺增生症中的潜力

Graves, Lara E; Christina, Sharntie; Mullany, Kathryn L; Alexander, Ian E; Falhammar, Henrik

Sleeping Beauty mRNA-LNP enables stable rAAV transgene expression in mouse and NHP hepatocytes and improves vector potency.

Sleeping Beauty mRNA-LNP 可在小鼠和 NHP 肝细胞中实现稳定的 rAAV 转基因表达,并提高载体效力

Zakas Philip M, Cunningham Sharon C, Doherty Ann, van Dijk Eva B, Ibraheim Raed, Yu Stephanie, Mekonnen Befikadu D, Lang Brendan, English Elizabeth J, Sun Gang, Duncan Miles C, Benczkowski Matthew S, Altshuler Robert C, Singh Malvenderjit Jagjit, Kibbler Emily S, Tonga Gulen Y, Wang Zi Jun, Wang Z Jane, Li Guangde, An Ding, Rottman James B, Bhavsar Yashvi, Purcell Cormac, Jain Rachit, Alberry Ryan, Roquet Nathaniel, Fu Yanfang, Citorik Robert J, Rubens Jacob R, Holmes Michael C, Cotta-Ramusino Cecilia, Querbes William, Alexander Ian E, Salomon William E

AAVolve: Concatenated long-read deep sequencing enables whole capsid tracking during shuffled AAV library selection

AAVolve:串联长读长深度测序技术可在AAV文库重组过程中实现全衣壳追踪

Scott, Suzanne; Westhaus, Adrian; Nazareth, Deborah; Cabanes-Creus, Marti; Navarro, Renina Gale; Chandra, Deborah; Zhu, Erhua; Venkateswaran, Aravind; Alexander, Ian E; Bauer, Denis C; Wilson, Laurence O W; Lisowski, Leszek

Gene therapy for urea cycle defects: An update from historical perspectives to future prospects

尿素循环缺陷的基因治疗:从历史回顾到未来展望

Duff, Claire; Alexander, Ian E; Baruteau, Julien