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
期刊:Blood Advances
影响因子:7.1
doi:10.1182/bloodadvances.2025018760
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