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Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene
GENETIC
2 trials
Sponsors
Genethon
Conditions
Wiskott-Aldrich Syndrome
Phase 1
Gene Therapy for Wiskott-Aldrich Syndrome (WAS)
Completed
NCT01347242
Genethon
Wiskott-Aldrich Syndrome
Start: 2011-03-31
End: 2019-11-13
Updated: 2021-06-03
Gene Therapy for WAS
Completed
NCT01347346
Genethon
Wiskott-Aldrich Syndrome
Start: 2011-05-31
End: 2017-01-09
Updated: 2018-05-22
Related Papers
Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome
Nature Medicine
2022-01-01
111 citations
Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult
Blood
2017-07-18
90 citations