1. The Lancet Haematology
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 
Francesca Ferrua, MD, Maria Pia Cicalese, MD, Stefania Galimberti, PhD, Stefania Giannelli, PhD, Francesca Dionisio, MSc, Federica Barzaghi, MD, Maddalena Migliavacca, MD, Maria Ester Bernardo, MD, Valeria Calbi, MD, Andrea Angelo Assanelli, MD, Marcella Facchini, PhD, Claudia Fossati, MSc, Elena Albertazzi, PhD, Samantha Scaramuzza, PhD, Immacolata Brigida, PhD, Serena Scala, PhD, Luca Basso-Ricci, MSc, Roberta Pajno, MD, Miriam Casiraghi, RN, Daniele Canarutto, MD, Federica Andrea Salerio, MSc, Prof Michael H Albert, MD, Antonella Bartoli, MScPharm, Prof Hermann M Wolf, MD, Rossana Fiori, MD, Paolo Silvani, MD, Salvatore Gattillo, MD, Anna Villa, MD, Luca Biasco, PhD, Christopher Dott, PhD, Emily J Culme-Seymour, PhD, Koenraad van Rossem, MD, Gillian Atkinson, PhD, Prof Maria Grazia Valsecchi, PhD, Prof Maria Grazia Roncarolo, MD, Prof Fabio Ciceri, MD, Prof Luigi Naldini, MD, Prof Alessandro Aiuti, MD 
The Lancet Haematology 
DOI: /S (19)
Copyright © 2019 The Author(s). Published by Elsevier Ltd. This is an Open Access article under the CC BY 4.0 license Terms and Conditions

2. Figure 1 Study design PBSC=peripheral blood stem cell.
The Lancet Haematology DOI: ( /S (19) )
Copyright © 2019 The Author(s). Published by Elsevier Ltd. This is an Open Access article under the CC BY 4.0 license Terms and Conditions

3. Figure 2
Multilineage engraftment of genetically corrected haemopoietic stem cells and peripheral blood cells in eight patients with Wiskott-Aldrich syndrome treated with lentiviral vector gene therapy
(A) In-vivo engraftment of transduced progenitor cells in bone marrow (LV-positive colonies). (B) Median VCN per cell in bone marrow cell lineages. (C) Median VCN per cell in peripheral blood cell lineages. No vector was detected in bone marrow total cells analysed before gene therapy. LV=lentiviral vector. VCN=vector copy number. GlyA=glycophorin A. NK=natural killer.
The Lancet Haematology DOI: ( /S (19) )
Copyright © 2019 The Author(s). Published by Elsevier Ltd. This is an Open Access article under the CC BY 4.0 license Terms and Conditions

4. Figure 3 Immune reconstitution and clinical benefit
Gene therapy was administered on day 1 (month 0). (A) Percentage of peripheral blood lymphocytes expressing WASP, as measured by flow cytometry. (B) T-cell proliferation response (expressed as SI) after incubation with increasing concentrations of anti-CD3i antigen, by observation period. Boxes represent upper and lower quartiles (outliers excluded). Horizontal line within the box is the median. Whiskers represent most extreme points ≤1·5 × IQR. Control data are from 12 healthy children. (C) Rate of severe infections (events per PYO) for each observation period. Error bars represent the 95% CI. (D) Number of days in hospital (days per PYO) for each observation period. Error bars represent the 95% CI. (E) Eczema scores are 1 (absent), 2 (mild), 3 (moderate), and 4 (severe). Six patients were followed up for at least 3 years after gene therapy. WASP=Wiskott-Aldrich syndrome protein. SI=stimulation index. Anti-CD3i=anti-immobilised CD3. PYO=patient-year of observation.
The Lancet Haematology DOI: ( /S (19) )
Copyright © 2019 The Author(s). Published by Elsevier Ltd. This is an Open Access article under the CC BY 4.0 license Terms and Conditions

5. Figure 4 Platelets and bleeding events
Gene therapy was administered on day 1 (month 0). (A) Percentage of platelets expressing WASP, as measured by flow cytometry. (B) Mean (SD) platelet count (× 109 cells per L). SD is not plotted for follow-up at 60 months because data were available for only two patients. (C) Moderate or severe bleeding events (per PYO) for each observation period. Error bars represent the 95% CI. (D) Distribution of all bleeding events for each observation period, by body site. Total number of events for each observation period is shown in the centre of each chart. WASP=Wiskott-Aldrich syndrome protein. PYO=patient-year of observation.
The Lancet Haematology DOI: ( /S (19) )
Copyright © 2019 The Author(s). Published by Elsevier Ltd. This is an Open Access article under the CC BY 4.0 license Terms and Conditions