FRIDAY, June 17th, 2022
08:30-09:00 - Gather together and on-site registration
09:00-09:15 - Introduction
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
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
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
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.
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
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
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
13:45 - 14:05 Ryan 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
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 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.
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 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
Chair: Michael Albert
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 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
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.
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