Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study.
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Ferrua F, et al. Among authors: albertazzi e.
Lancet Haematol. 2019 May;6(5):e239-e253. doi: 10.1016/S2352-3026(19)30021-3. Epub 2019 Apr 10.
Lancet Haematol. 2019.
PMID: 30981783
Free PMC article.
Clinical Trial.