Ligand-modified rAAV6 vectors with nanoblades allow high-level gene knockin in HSPCs without compromising cell survival
带有纳米刀片的配体修饰的rAAV6载体可在不影响细胞存活率的情况下，实现造血干细胞（HSPC）中的高水平基因敲入。
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1016/j.omtn.2025.102495
Gutierrez-Guerrero, Alejandra; Périan, Séverine; Leray, Aurélien; Martinello, Chiara; Abrey Recalde, Maria Jimena; Costa, Caroline; Herrero, Cecilia Iglesias; Bouzelha, Mohammed; Alvarez-Dorta, Dimitri; Gouin, Sébastien G; Ayuso, Eduard; Adjali, Oumeya; Büning, Hildegard; Deniaud, David; Mével, Mathieu; Verhoeyen, Els
