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

TNFα signaling restores steady-state hematopoiesis in a TNFαKO mouse model of anemia of inflammation

在炎症性贫血的TNFαKO小鼠模型中,TNFα信号通路可恢复稳态造血功能。

Guerra, Amaliris; Lo Presti, Vania; Chen, Ding-Wen; Martins, Ana Catarina; Rivera, Ariel; Hamilton, Nolan; Sharma, Pankja; Ginzburg, Yelena Z; Castruccio Castracani, Carlo; Casu, Carla; Gupta, Ritama; Gozzelino, Raffaella; Behrens, Edward M; Bennett, Laura F; Paulson, Robert F; Kurre, Peter; Rivella, Stefano

An erythroid-specific lentiviral vector improves anemia and iron metabolism in a new model of XLSA

一种红细胞特异性慢病毒载体可改善XLSA新模型中的贫血和铁代谢。

Castruccio Castracani, Carlo; Breda, Laura; Papp, Tyler E; Guerra, Amaliris; Radaelli, Enrico; Assenmacher, Charles-Antoine; Finesso, Giovanni; Mui, Barbara L; Tam, Ying K; Fontana, Simona; Riganti, Chiara; Fiorito, Veronica; Petrillo, Sara; Tolosano, Emanuela; Parhiz, Hamideh; Rivella, Stefano

Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrations

利用最小的慢病毒基因组整合实现对异染性脑白质营养不良的有效基因治疗

Lucas Tricoli ,Sunetra Sase ,Julia L Hacker ,Vi Pham ,Maxwell Chappell ,Laura Breda ,Stephanie N Hurwitz ,Naoto Tanaka ,Carlo Castruccio Castracani ,Amaliris Guerra ,Zhongqi Hou ,Lars Schlotawa ,Karthikeyan Radhakrishnan ,Matthew Hogenauer ,Aoife Roche ,John Everett ,Frederic Bushman ,Peter Kurre ,Rebecca Ahrens-Nicklas ,Laura A Adang ,Adeline L Vanderver ,Stefano Rivella

Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapy

利用靶向造血干细胞的脂质纳米颗粒构建致死性α-地中海贫血小鼠模型,并通过慢病毒基因治疗进行治疗。

Chappell, Maxwell E; Breda, Laura; Tricoli, Lucas; Guerra, Amaliris; Jarocha, Danuta; Castruccio Castracani, Carlo; Papp, Tyler E; Tanaka, Naoto; Hamilton, Nolan; Triebwasser, Michael P; Ghiaccio, Valentina; Fedorky, Megan T; Gollomp, Kandace L; Bochenek, Veronica; Roche, Aoife M; Everett, John K; Cook, Emma J; Bushman, Frederic D; Teawtrakul, Nattiya; Glentis, Stavros; Kattamis, Antonis; Mui, Barbara L; Tam, Ying K; Weissman, Drew; Abdulmalik, Osheiza; Parhiz, Hamideh; Rivella, Stefano

Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiency

造血干细胞基因疗法改善了具有临床意义的多发性硫酸酯酶缺乏症小鼠模型的预后。

Vi Pham ,Lucas Tricoli ,Xinying Hong ,Parith Wongkittichote ,Carlo Castruccio Castracani ,Amaliris Guerra ,Lars Schlotawa ,Laura A Adang ,Amanda Kuhs ,Margaret M Cassidy ,Owen Kane ,Emily Tsai ,Maximiliano Presa ,Cathleen Lutz ,Stefano B Rivella ,Rebecca C Ahrens-Nicklas

Combination of a TGF-β ligand trap (RAP-GRL) and TMPRSS6-ASO is superior for correcting β-thalassemia.

TGF-β配体陷阱(RAP-GRL)与TMPRSS6-ASO的组合在纠正β-地中海贫血方面更胜一筹

Guerra Amaliris, Hamilton Nolan, Rivera Ariel, Demsko Perry, Guo Shuling, Rivella Stefano

Novel potential therapeutics to modify iron metabolism and red cell synthesis in diseases associated with defective erythropoiesis

新型潜在疗法可用于改善与红细胞生成缺陷相关的疾病中的铁代谢和红细胞合成

Guerra, Amaliris; Parhiz, Hamideh; Rivella, Stefano

Inclusion of a short hairpin RNA targeting BCL11A into a β-globin expressing vector allows concurrent synthesis of curative adult and fetal hemoglobin

将针对 BCL11A 的短发夹 RNA 纳入 β-珠蛋白表达载体,可以同时合成治疗性成人血红蛋白和胎儿血红蛋白

Silvia Pires Lourenco, Danuta Jarocha, Valentina Ghiaccio, Amaliris Guerra, Osheiza Abdulmalik, Ping La, Alexandra Zezulin, Kim Smith-Whitley, Janet L Kwiatkowski, Virginia Guzikowski, Yukio Nakamura, Tobias Raabe, Laura Breda, Stefano Rivella

Effect of hyaluronic acid incorporation method on the stability and biological properties of polyurethane-hyaluronic acid biomaterials

透明质酸掺入方法对聚氨酯-透明质酸生物材料稳定性和生物学性能的影响

Ruiz, Amaliris; Rathnam, Kashmila R; Masters, Kristyn S

Transient photoreceptor deconstruction by CNTF enhances rAAV-mediated cone functional rescue in late stage CNGB3-achromatopsia

CNTF介导的瞬时感光细胞解体增强了rAAV介导的视锥细胞功能恢复,从而改善晚期CNGB3致全色盲。

Komáromy, András M; Rowlan, Jessica S; Corr, Amanda T Parton; Reinstein, Shelby L; Boye, Sanford L; Cooper, Ann E; Gonzalez, Amaliris; Levy, Britt; Wen, Rong; Hauswirth, William W; Beltran, William A; Aguirre, Gustavo D