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

Allele-selective disruption of pathogenic VWF variants in type 2 von Willebrand disease using CRISPR/Cas9

利用 CRISPR/Cas9 技术对 2 型血管性血友病中的致病性 VWF 变异体进行等位基因选择性破坏

Bär, Isabel; Groten, Stijn A; Barraclough, Alastair; Bürgisser, Petra E; van Kwawegen, Calvin; Lenting, Peter J; van Moort, Iris; Eikenboom, Jeroen C J; Leebeek, Frank W G; Voorberg, Jan; van den Biggelaar, Maartje; Bierings, Ruben

A red blood cell-based antigen delivery system to facilitate T cell epitope presentation to promote peripheral tolerance to ADAMTS13 in immune-mediated thrombotic thrombocytopenic purpura

基于红细胞的抗原递送系统,可促进T细胞表位呈递,从而在免疫介导的血栓性血小板减少性紫癜中促进对ADAMTS13的外周耐受。

Dierickx, Margot; Groten, Stijn; Miranda, Mariarosaria; Langerhorst, Pieter; van der Zwaan, Carmen; Mul, Erik; Hoogendijk, Arie J; Voorberg, Jan

Hemostatic and platelet proteins are associated with bleeding grade and time to major bleed in hemato-oncological patients

血液肿瘤患者的止血蛋白和血小板蛋白与出血程度和发生大出血的时间相关。

Muñoz Sandoval, Diana; Smit, Eva R; Rojas-Rodriguez, Felipe; van der Meer, Pieter F; Voorberg, Jan; van der Zwaan, Carmen; Langerhorst, Pieter; Hoogendijk, Arie J; van Dam, Teunis J P; Kerkhoffs, Jean-Louis; Ypma, Paula F; van den Biggelaar, Maartje

FVIII peptides presented on HLA-DP and identification of an A3 domain peptide binding with high affinity to the commonly expressed HLA-DP4

HLA-DP 呈递的 FVIII 肽段以及与常见表达的 HLA-DP4 高亲和力结合的 A3 结构域肽段的鉴定

Miranda, Mariarosaria; Hansen, Bjarke Endel; Wehbi, Batoul; Porcheddu, Valeria; Van Alphen, Floris P J; Kaijen, Paul; Fijnvandraat, Karin; Lacroix-Desmazes, Sebastien; Van den Biggelaar, Maartje; Maillere, Bernard; Voorberg, Jan; Consortium, Educ

Amelioration of a von Willebrand disease type 2B phenotype in vivo upon treatment with allele-selective siRNAs.

用等位基因选择性siRNA治疗后,体内血管性血友病2B型表型得到改善

Linthorst Noa A, Jongejan Yvonne K, Dirven Richard J, Laan Sebastiaan N J, Bierings Ruben, Casari Caterina, Cordfunke Robert A, Dahlman James E, Dolezal Natasja, Drijfhout Jan Wouter, Leebeek Frank W G, Ruhaak L Renee, Schrader Echeverri Elisa, Voorberg Jan, van Vlijmen Bart J M, Denis Cécile V, Eikenboom Jeroen C J

Rational design of FVIII sialylated peptides to target Siglec-3 and Siglec-9 and improve peptide formulations for reverse vaccines.

合理设计 FVIII 唾液酸化肽以靶向 Siglec-3 和 Siglec-9,并改进反向疫苗的肽制剂

Nardini Eleonora, Keumatio Doungstop Brigitte-Carole, Gerpe-Amor Tania, de Haas Aram M, de Kok Mike, Peterse Evert, Kalay Hakan, Li Rui-Jún E, Chiodo Fabrizio, Silipo Alba, Voorberg Jan, van Kooyk Yvette

Sustained high expression of human FVII following AAV8-mediated gene delivery in mice.

在小鼠体内通过 AAV8 介导的基因递送实现人 FVII 的持续高表达

D'Amico Andrea, Blits Bas, van den Herik Joëlle, de Winter Fred, Kaijen Paul H P, Hobo Barbara, van Asbeck Alexander Henrik, Verhaagen Joost, Mosmuller David G M, Voorberg Jan

Correction: Rewriting the script: gene therapy and genome editing for von Willebrand Disease

更正:重写剧本:基因疗法和基因组编辑治疗血管性血友病

Barraclough, Alastair; Bär, Isabel; van Duijl, Tirsa; Fijnvandraat, Karin; Eikenboom, Jeroen C J; Leebeek, Frank W G; Bierings, Ruben; Voorberg, Jan; Trasanidou, Despoina

Rewriting the script: gene therapy and genome editing for von Willebrand Disease

改写剧本:基因疗法和基因组编辑治疗血管性血友病

Barraclough, Alastair; Bär, Isabel; van Duijl, Tirsa; Fijnvandraat, Karin; Eikenboom, Jeroen C J; Leebeek, Frank W G; Bierings, Ruben; Voorberg, Jan; Trasanidou, Despoina

Large deletions in the F8 gene predict immune tolerance induction failure in people with severe hemophilia A

F8基因的大片段缺失预示着重度A型血友病患者免疫耐受诱导失败。

Oomen, Ilja; Abdi, Amal; Broer, Linda; Camelo, Ricardo M; Callado, Fábia M R A; Carvalho, Luany E M; Calcaterra, Ilenia L; Carcao, Manuel; Castaman, Giancarlo; Eikenboom, Jeroen C J; Fischer, Kathelijn; Franco, Vivian K B; Geissler, Judy; Kuijpers, Taco W; Leebeek, Frank W G; Lillicrap, David; Lorenzato, Cláudia S; Mancuso, Maria Elisa; Matino, Davide; Di Minno, Matteo N D; Mo, Aomei; Mohseny, Alex B; Nagelkerke, Sietse Q; Oldenburg, Johannes; Rezende, Suely Meireles; Rivard, Georges-Etienne; Rydz, Natalia; Schols, Saskia E M; Tanck, Michael W T; Voorberg, Jan; Fijnvandraat, Karin; Gouw, Samantha C