​09:00-09:15 - Introduction

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Sumathi Iyengar- Amir Kedar, WAS organizations and Michael Albert, Munich, Germany

​09:15- 09:45 - Plenary session A 
Anna Villa  - San Raffaele Telethon Institute for Gene Therapy,  Milano & IRGB, National Research Council.
"Platelets defects in Wiskott-Aldrich Syndrome"

​RESEARCH SESSION 1 

Chair: Adrian Thrasher

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09:45 - 10:00 Rhaissa Vieira - Department of Microbiology Tumor and Cell Biology, Karolinska Institutet, Sweden.

Flow Cytometry-based Drug Screening for stabilization of WASp to restore Megakaryocyte and Platelet Function

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10:00 - 10:15 Jordan Chill - Department of Chemistry, Bar Ilan University, Israel.   

Towards a Molecular Understanding of WAS/XLT Using Biological Nuclear Magnetic Resonance

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10:15 - 10:30 Roberta D'aulerio - Department of Microbiology Tumor and Cell Biology, Karolinska Institutet, Sweden.

Revealing the molecular role of WASp in the nucleus of B cells.

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10:30 - 10:45 Questions & answers and discussion  

10:45 - 11:05 - Break

​11:05- 11:35 - Plenary session B
David J. Rawlings - Director, Center for immunity and immunotherapies, Seattle Children’s Research Institute, USA
“Lessons learned regarding immune tolerance and progress towards new therapies for WAS”

​RESEARCH SESSION 2 

Chair: Lisa Westerberg

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11:35 - 11:50 Minghui He - Department of Microbiology Tumor and Cell Biology, Karolinska Institutet, Sweden.

Constitutively active WASp in X-linked neutropenia leads to compromised B cell division and accelerated plasma cell differentiation

11:50 - 12:05 Lia Pinho - Department of Microbiology Tumor and Cell Biology, Karolinska Institutet, Sweden.

A novel Arg431Trp mutation of WASp causes an intermediate WAS and XLN phenotype

12:05 - 12:15 Questions & answers and discussion

GENE THERAPY SESSION

Chair: Anna Villa

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12:15 - 12:45  Francesca Ferrua - San Raffaele Telethon Institute for Gene Therapy, Italy.

Hematopoietic Stem and Progenitor Cell Lentiviral Gene Therapy for Wiskott-Aldrich Syndrome: Up to 10.5 Years of Follow-Up in 17 Subjects

12:45- 13:45- Lunch

GENE THERAPY SESSION (cont.)

Chair: Anna Villa

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13:45 - 14:05 Rayan Wong (Invited speaker) – ImmunoVec, CA, USA.

Bioinformatic-Guided Design of a Lentiviral Vector for Wiskott-Aldrich Syndrome Recapitulates Endogenous WAS Gene Expression

14:05 - 14:20  Maria Pia Cicalese - Clinical Research Institute (CRU), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Restoration of follicular T cells in patients with Wiskott-Aldrich Syndrome after gene therapy

14:20 – 14:35 Melissa Pille – Department of Diagnostic Sciences, Ghent University, Belgium. 

CRISPR/Cas9- mediated gene editing for treatment of the Wiskott-Aldrich Syndrome

14:35 - 14:50  Questions & answers and discussion

CLINICAL SESSION 1

Chair: Fabio Candotti

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14:50 - 15:05 Annarosa Soresina – Pediatrics Clinic, ASST Spedali Civili of Brescia, Univ. of Brescia, Italy.

Long term outcome in Wiskott- Aldrich Syndrome (WAS) and X-Linked Thrombocytopenia (XLT): a prospective multicenter national study.

15:05 - 15:20 - TBC

15:20 - 15:35 Charline Miot - University Hospital of Angers, France.

Long-term Outcome of Mild WAS/XLT Patients: Experience from the Registry of the French National Reference Center for Primary Immunodeficiencies (CEREDIH)

15:35 - 15:50  Questions & answers and discussion

15:50 - 16:10 - Break

16:10 - 16:40 - Plenary session C

Michael Albert - Head of the pediatric stem cell transplantation unit at the Dr. von Haunersches University Children’s Hospital, Munich

"HSCT for WAS – What have we learned in 50 years and what promises does the future hold?"

CLINICAL SESSION 2

Chair: Michael Albert

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16:40 - 17:00 Carmem Bonfim (Invited speaker) – Director Pediatric Blood and Marrow Transplantation Program, Federal University of Paraná, Curitiba, Brazil.

The challenges and hurdles to improve outcomes after HSCT for patients with Wiskott Aldrich syndrome in restricted resources countries

17:00 - 17:15 Samuele Naviglio - Department of Pediatric Hematology-Oncology, Institute for Maternal and Child Health IRCCS "Burlo Garofolo", Trieste, Italy.

Autoinflammatory manifestations and response to treatment with anti-interleukin-1 agents in patients with Wiskott-Aldrich Syndrome

17:15 - 17:30 Shanmuganathan Chandrakasan – Emory University, Department of Pediatrics, Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta

Clinical spectrum of Carriers of X-linked Wiskott Aldrich syndrome gene: Self-reported survey of 193 carriers

17:30 - 17:45  Questions & answers and discussion

​17:45-18:00 - Closing discussions and summary of the day

Adrian Thrasher, Michael Albert

18:00 - 19:00 -  Reception

An informal get-together to renew acquaintances and meet new colleagues will be held at the Meeting Venue. All registered participants are invited to join.