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

DNA delivered by lipid nanoparticles induces CD8+ T cell-dependent antitumor responses and enhances anti-PD-L1 therapy.

通过脂质纳米颗粒递送的DNA可诱导CD8+ T细胞依赖性抗肿瘤反应,并增强抗PD-L1疗法。

Yum Seoyun, Rodríguez-Garcia Alba, Castellsagué Joan, Giménez-Alejandre Marta, Colell Guillem, Colell Salut, Lobo-Jarne Teresa, LaRue Mark A, Minnier Michael A, Yazicioglu Mustafa N, Zhang Rui, Anguela Xavier M, Nahvi Ali, Walsh Matthew C, Armour Sean M, Guedan Sonia, Cejas Pedro J

ARI0003: Co-transduced CD19/BCMA dual-targeting CAR-T cells for the treatment of non-Hodgkin lymphoma

ARI0003:共转导的CD19/BCMA双靶向CAR-T细胞用于治疗非霍奇金淋巴瘤

Bachiller, Mireia; Barceló-Genestar, Nina; Rodriguez-Garcia, Alba; Alserawan, Leticia; Dobaño-López, Cèlia; Giménez-Alejandre, Marta; Castellsagué, Joan; Colell, Salut; Otero-Mateo, Marc; Antoñana-Vildosola, Asier; Español-Rego, Marta; Ferruz, Noelia; Pascal, Mariona; Martín-Antonio, Beatriz; Anguela, Xavier M; Fillat, Cristina; Olesti, Eulàlia; Calvo, Gonzalo; Juan, Manel; Delgado, Julio; Pérez-Galán, Patricia; Urbano-Ispizua, Álvaro; Guedan, Sonia

Hemophilia B and gene therapy: a new chapter with etranacogene dezaparvovec

B型血友病与基因治疗:etranacogene dezaparvovec开启新篇章

Anguela, Xavier M; High, Katherine A

Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A

对小鼠和非人类灵长类动物中优化的 AAV-FVIII 载体用于治疗血友病 A 的临床前评估

Liron Elkouby, Sean M Armour, Raffaella Toso, Marti DiPietro, Robert J Davidson, Giang N Nguyen, Mallory Willet, Stephanie Kutza, Joseph Silverberg, Jennifer Frick, Marco Crosariol, Yuhuan Wang, Chuansong Wang, Katherine A High, Denise E Sabatino, Xavier M Anguela

Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B

首次人体血管内递送腺相关病毒(AAV)进行基因转移的长期随访:AAV2-hFIX16 用于治疗重型血友病 B

George, Lindsey A; Ragni, Margaret V; Rasko, John E J; Raffini, Leslie J; Samelson-Jones, Benjamin J; Ozelo, Margareth; Hazbon, Maria; Runowski, Alexa R; Wellman, Jennifer A; Wachtel, Katie; Chen, Yifeng; Anguela, Xavier M; Kuranda, Klaudia; Mingozzi, Federico; High, Katherine A

Experimental Variables that Affect Human Hepatocyte AAV Transduction in Liver Chimeric Mice

影响肝嵌合小鼠中人肝细胞AAV转导的实验变量

Zou, Chenhui; Vercauteren, Koen O A; Michailidis, Eleftherios; Kabbani, Mohammad; Zoluthkin, Irene; Quirk, Corrine; Chiriboga, Luis; Yazicioglu, Mustafa; Anguela, Xavier M; Meuleman, Philip; High, Katherine A; Herzog, Roland W; de Jong, Ype P

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

利用高比活性因子IX变体治疗B型血友病

George, Lindsey A; Sullivan, Spencer K; Giermasz, Adam; Rasko, John E J; Samelson-Jones, Benjamin J; Ducore, Jonathan; Cuker, Adam; Sullivan, Lisa M; Majumdar, Suvankar; Teitel, Jerome; McGuinn, Catherine E; Ragni, Margaret V; Luk, Alvin Y; Hui, Daniel; Wright, J Fraser; Chen, Yifeng; Liu, Yun; Wachtel, Katie; Winters, Angela; Tiefenbacher, Stefan; Arruda, Valder R; van der Loo, Johannes C M; Zelenaia, Olga; Takefman, Daniel; Carr, Marcus E; Couto, Linda B; Anguela, Xavier M; High, Katherine A

Adeno-associated viral vectors for the treatment of hemophilia

腺相关病毒载体用于治疗血友病

High, Katherine A; Anguela, Xavier M

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

体内白蛋白基因座基因组编辑作为蛋白质替代疗法的平台

Rajiv Sharma, Xavier M Anguela, Yannick Doyon, Thomas Wechsler, Russell C DeKelver, Scott Sproul, David E Paschon, Jeffrey C Miller, Robert J Davidson, David Shivak, Shangzhen Zhou, Julianne Rieders, Philip D Gregory, Michael C Holmes, Edward J Rebar, Katherine A High

Robust ZFN-mediated genome editing in adult hemophilic mice

成年嗜血病小鼠中 ZFN 介导的稳健基因组编辑

Xavier M Anguela, Rajiv Sharma, Yannick Doyon, Jeffrey C Miller, Hojun Li, Virginia Haurigot, Michelle E Rohde, Sunnie Y Wong, Robert J Davidson, Shangzhen Zhou, Philip D Gregory, Michael C Holmes, Katherine A High