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

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 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

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

Innate functions of immunoglobulin M lessen liver gene transfer with helper-dependent adenovirus.

免疫球蛋白 M 的固有功能可减少辅助依赖性腺病毒介导的肝脏基因转移

Unzu Carmen, Melero Ignacio, Morales-Kastresana Aizea, Sampedro Ana, Serrano-Mendioroz Irantzu, Azpilikueta Arantza, Ochoa María Carmen, Dubrot Juan, Martínez-Ansó Eduardo, Fontanellas Antonio

Targeting HIV Gag p24 to DICR on dendritic cells induces T cell and potent and long-lasting antibody responses in non-human primates

将 HIV Gag p24 靶向树突状细胞上的 DICR 可诱导非人灵长类动物产生 T 细胞反应以及强效且持久的抗体反应。

Córdoba, Karol M; Jericó, Daniel; Jiang, Lei; Collantes, María; Alegre, Manuel; García-Ruiz, Leyre; Manzanilla, Oscar; Sampedro, Ana; Herranz, Jose M; Insausti, Iñigo; Martinez de la Cuesta, Antonio; Urigo, Francesco; Alcaide, Patricia; Morán, María; Martín, Miguel A; Lanciego, José Luis; Lefebvre, Thibaud; Gouya, Laurent; Quincoces, Gemma; Unzu, Carmen; Hervas-Stubbs, Sandra; Falcón-Pérez, Juan M; Alegre, Estíbaliz; Aldaz, Azucena; Fernández-Seara, María A; Peñuelas, Iván; Berraondo, Pedro; Martini, Paolo G V; Avila, Matias A; Fontanellas, Antonio; Banerjee, K; Balsitis, S; Jamil, T; Jones, C; Dey, A; Flandez, J; Brito, L; Cu, Y; Beard, C; Santra, S; Pal, R; Miller, N; Valiante, NM; Mason, P; Barnett, SW; Otten, GR; Flamar, A; Le Grand, R; Contreras, V; Zurawski, S; Dereuddre-Bosquet, N; Mangeot, I; Martinon, F; Oh, S; Banchereau, J; Zurawski, G; Levy, Y

Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates

短暂和强效的药物免疫抑制未能改善非人灵长类动物中基于AAV的肝脏基因转移。

Unzu, Carmen; Hervás-Stubbs, Sandra; Sampedro, Ana; Mauleón, Itsaso; Mancheño, Uxua; Alfaro, Carlos; de Salamanca, Rafael Enríquez; Benito, Alberto; Beattie, Stuart G; Petry, Harald; Prieto, Jesús; Melero, Ignacio; Fontanellas, Antonio

Intensive pharmacological immunosuppression allows for repetitive liver gene transfer with recombinant adenovirus in nonhuman primates

通过强化药物免疫抑制,可以在非人灵长类动物中利用重组腺病毒进行重复的肝脏基因转移。

Fontanellas, Antonio; Hervás-Stubbs, Sandra; Mauleón, Itsaso; Dubrot, Juan; Mancheño, Uxua; Collantes, María; Sampedro, Ana; Unzu, Carmen; Alfaro, Carlos; Palazón, Asis; Smerdou, Cristian; Benito, Alberto; Prieto, Jesús; Peñuelas, Iván; Melero, Ignacio