PRESENTATIONS  SUMMARIES. 

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​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 

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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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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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.

​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 

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

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

GENE THERAPY SESSION

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

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Maria Carmina Castiello - San Raffaele Telethon Institute for Gene Therapy, IRGB National Research Council , Italy.

B cell reconstitution in WAS patients after Gene Therapy 

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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

GENE THERAPY SESSION (cont.)

Ryan Wong (Invited speaker) – ImmunoVec, CA, USA.

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

Melissa Pille – Department of Diagnostic Sciences, Ghent University, Belgium. 

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

GENE THERAPY PANEL

Chair: Michael Albert

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14:45 - 15:15 Anne Galy, Fabio Candotti, Anna Villa, David Rawlings, Francesca Ferrua, Suhag Parikh 

"Future of Gene Therapy for WAS: Challenges and Opportunities" 

CLINICAL SESSION 1

Raffaele Badolato –  Universita degli Studi di Brescia, Italy.

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

Deepti Suri – Allergy and Immunology Unit, Department of Pediatrics, Postgraduate Institute of Medical Education and Research, Chandigarh, India

Wiskott Aldrich syndrome: 15 years’ experience at Post Graduate Institute of Medical Education and Research, Chandigarh, India.

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)

16:25 - 16:55 - Plenary session C

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

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

CLINICAL SESSION 2

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

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

Suhag Parikh – 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