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

Orthogonal characterization of rAAV reveals vector attributes that drive ITR repair, self-complementary genome formation, and transgene expression.

rAAV 的正交表征揭示了驱动 ITR 修复、自我互补基因组形成和转基因表达的载体属性。

Llinares Carla, Namkung Suk, Yip Mitchell, Vales África, Olagüe Cristina, Mauleón Itsaso, Isola Sergio, Moeini Pedram, González-Aseguinolaza Gloria, Tai Phillip W L, Unzu Carmen

Clinical perspective: Advancing hemophilia treatment through gene therapy approaches

临床视角:通过基因治疗方法推进血友病治疗

Thornburg, Courtney D; Pipe, Steve W; Cantore, Alessio; Unzu, Carmen; Jones, Micheala; Miesbach, Wolfgang A

Endocardial-to-mesenchymal transition underlies cardiac outflow tract septation and bicuspid aortic valve formation in the Syrian hamster model.

在叙利亚仓鼠模型中,心内膜间质转化是心脏流出道隔膜形成和二叶式主动脉瓣形成的基础

Soto-Navarrete María Teresa, Pozo-Vilumbrales Bárbara, López-Unzu Miguel Á, Martín-Chaves Laura, Durán Ana C, Fernández Borja

Development of a novel gene editing lexicon for hemophilia: methodology and results

开发用于血友病的新型基因编辑词汇表:方法和结果

Kessler, Craig M; Valentino, Leonard A; Thornburg, Courtney D; Unzu, Carmen; Kay, Mark A; Peyvandi, Flora; Smith, Penni; Miesbach, Wolfgang; McKeown, William; Pierce, Glenn F; Khair, Kate; Starcevic, Katarina; Pillai, Monisha; Jones, Micheala; Sindhurakar, Anil; Whyte, Lauren; Delwart, Virginie; Chiao, Megan; Gutstein, David E; Antonino, Ilia; Hermans, Cedric; Pipe, Steven W

Harnessing whole human liver ex situ normothermic perfusion for preclinical AAV vector evaluation

利用全人肝脏离体常温灌注进行临床前 AAV 载体评估

Marti Cabanes-Creus, Sophia H Y Liao, Renina Gale Navarro, Maddison Knight, Deborah Nazareth, Ngee-Soon Lau, Mark Ly, Erhua Zhu, Ramon Roca-Pinilla, Ricardo Bugallo Delgado, Ana F Vicente, Grober Baltazar, Adrian Westhaus, Jessica Merjane, Michael Crawford, Geoffrey W McCaughan, Carmen Unzu, Gloria

Harnessing gene therapy for liver metabolic dysfunction: An innovative approach to MASH treatment

利用基因疗法治疗肝脏代谢功能障碍:MASH治疗的一种创新方法

Unzu, Carmen; Fernández-Barrena, Maite G

Enhanced AAV transduction across preclinical CNS models: A comparative study in human brain organoids with cross-species evaluations

增强 AAV 在临床前 CNS 模型中的转导:对人类脑类器官进行跨物种评估的比较研究

Matthieu Drouyer, Jessica Merjane, Teodora Nedelkoska, Adrian Westhaus, Suzanne Scott, Scott Lee, Peter G R Burke, Simon McMullan, Jose L Lanciego, Ana F Vicente, Ricardo Bugallo, Carmen Unzu, Gloria González-Aseguinolaza, Anai Gonzalez-Cordero, Leszek Lisowski

A novel gene editing lexicon strategy for the haemophilia community: Research plan for development and preliminary results

针对血友病群体的新型基因编辑词汇策略:研发计划及初步结果

Hermans, Cedric; Valentino, Leonard A; Thornburg, Courtney D; Unzu, Carmen; Kay, Mark A; Peyvandi, Flora; Smith, Penni; Miesbach, Wolfgang; McKeown, William; Pierce, Glenn F; Khair, Kate; Pipe, Steven W; Starcevic, Katarina; Pillai, Monisha; Jones, Micheala; Chiao, Megan; Antonino, Ilia; Kessler, Craig

Topoisomerase Inhibitors Increase Episomal DNA Expression by Inducing the Integration of Episomal DNA in Hepatic Cells

拓扑异构酶抑制剂通过诱导肝细胞中游离 DNA 的整合来增加游离 DNA 的表达

Andoni Gómez-Moreno, Enara San Sebastian, Jennifer Moya, Pilar Gomollón-Zueco, Sergio Isola, África Vales, Gloria González-Aseguinolaza, Carmen Unzu, Urtzi Garaigorta

Choice of vector and surgical approach enables efficient cochlear gene transfer in nonhuman primate

载体和手术方法的选择可实现非人类灵长类动物耳蜗基因的有效转移

Eva Andres-Mateos #, Lukas D Landegger #, Carmen Unzu #, Jean Phillips, Brian M Lin, Nicholas A Dewyer, Julio Sanmiguel, Fotini Nicolaou, Michelle D Valero, Kathrin I Bourdeu, William F Sewell, Rudolph J Beiler, Michael J McKenna, Konstantina M Stankovic, Luk H Vandenberghe