Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models.
基因疗法治疗威斯科特-奥尔德里奇综合征的临床前模型长期疗效和安全性证据
期刊:Molecular Therapy
影响因子:12
doi:10.1038/mt.2009.31
Marangoni Francesco, Bosticardo Marita, Charrier Sabine, Draghici Elena, Locci Michela, Scaramuzza Samantha, Panaroni Cristina, Ponzoni Maurilio, Sanvito Francesca, Doglioni Claudio, Liabeuf Marie, Gjata Bernard, Montus Marie, Siminovitch Katherine, Aiuti Alessandro, Naldini Luigi, Dupré Loïc, Roncarolo Maria Grazia, Galy Anne, Villa Anna
