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

Balancing Promise and Peril: Hemophilia Gene Therapy Insights

权衡希望与风险:血友病基因疗法的启示

Akula, Saicharan; Borroni, Ester; Cottonaro, Alessia; Follenzi, Antonia; Merlin, Simone

Factor VIII promotes angiogenesis and vessel stability regulating extracellular matrix proteins

因子VIII通过调节细胞外基质蛋白促进血管生成和血管稳定性。

Olgasi, Cristina; Cucci, Alessia; Molineris, Ivan; Assanelli, Simone; Anselmi, Francesca; Borsotti, Chiara; Sgromo, Chiara; Lauria, Andrea; Merlin, Simone; Walker, Gillian E; Oliviero, Salvatore; Follenzi, Antonia

Immune tolerance promotion by LSEC-specific lentiviral vector-mediated expression of the transgene regulated by the stabilin-2 promoter

通过 LSEC 特异性慢病毒载体介导的转基因表达促进免疫耐受,该转基因受 stabilin-2 启动子调控

Borroni, Ester; Borsotti, Chiara; Cirsmaru, Roberta A; Kalandadze, Vakhtang; Famà, Rosella; Merlin, Simone; Brown, Brian; Follenzi, Antonia

Therapeutic potential of fetal liver cell transplantation in hemophilia A mice

胎肝细胞移植治疗A型血友病小鼠的潜力

Merlin, Simone; Akula, Saicharan; Cottonaro, Alessia; Garcia-Leal, Tamara; Serrano, Luis Javier; Borroni, Ester; Kalandadze, Vakhtang; Galiano, Rocio; Borsotti, Chiara; Liras, Antonio; Sanchez, María José; Follenzi, Antonia

Factor VIII as a potential player in cancer pathophysiology

因子VIII作为癌症病理生理学中的潜在参与者

Walker, Gillian E; Merlin, Simone; Zanolini, Diego; Vandoni, Andrea; Volpe, Alessandro; Gaidano, Gianluca; Valente, Guido; Olivero, Martina; Follenzi, Antonia

Dissection of pleiotropic effects of variants in and adjacent to F8 exon 19 and rescue of mRNA splicing and protein function

对F8外显子19及其邻近区域变异的多效性效应进行剖析,并探讨其对mRNA剪接和蛋白质功能的恢复作用

Lombardi, Silvia; Leo, Gabriele; Merlin, Simone; Follenzi, Antonia; McVey, John H; Maestri, Iva; Bernardi, Francesco; Pinotti, Mirko; Balestra, Dario

Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device

利用植入式装置中慢病毒载体转导的BOECs高效安全地纠正A型血友病

Olgasi, Cristina; Borsotti, Chiara; Merlin, Simone; Bergmann, Thorsten; Bittorf, Patrick; Adewoye, Adeolu Badi; Wragg, Nicholas; Patterson, Kelcey; Calabria, Andrea; Benedicenti, Fabrizio; Cucci, Alessia; Borchiellini, Alessandra; Pollio, Berardino; Montini, Eugenio; Mazzuca, Delfina M; Zierau, Martin; Stolzing, Alexandra; Toleikis, Philip M; Braspenning, Joris; Follenzi, Antonia

Escape or Fight: Inhibitors in Hemophilia A

逃避还是反抗:血友病A中的抑制剂

Merlin, Simone; Follenzi, Antonia

Regulatory-Compliant Validation of a Highly Sensitive qPCR for Biodistribution Assessment of Hemophilia A Patient Cells

符合监管要求的用于血友病A患者细胞生物分布评估的高灵敏度qPCR验证

Bittorf, Patrick; Bergmann, Thorsten; Merlin, Simone; Olgasi, Cristina; Pullig, Oliver; Sanzenbacher, Ralf; Zierau, Martin; Walles, Heike; Follenzi, Antonia; Braspenning, Joris

FVIII expression by its native promoter sustains long-term correction avoiding immune response in hemophilic mice

FVIII通过其自身启动子表达,可维持长期矫正作用,避免血友病小鼠产生免疫反应。

Merlin, Simone; Famà, Rosella; Borroni, Ester; Zanolini, Diego; Bruscaggin, Valentina; Zucchelli, Silvia; Follenzi, Antonia