​08:30- 09:00 Gather together and On-site Registration
​09:00- 09:10 Welcome and Introduction
Sumathi Iyengar & Amir Kedar, WAS organizations and Anna Villa, IRCCS Ospedale San Raffaele, Milan, Italy.
​09:10- 09:20 Family Story
Gene Therapy Family Story - Valentina Pagani, Rosa Giglia.
​09:20- 09:50 Plenary session A
Alessandro Aiuti - head of the Pediatric Immunohematology Unit at the IRCCS San Raffaele, Milano, Italy.
"The long path of gene therapy for Wiskott-Aldrich Syndrome"
09:50 - 10:35 Gene Therapy Session
Chair: Fabio Candotti
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09:50 - 10:05 Francesca Ferrua - San Raffaele Telethon Institute for Gene Therapy, Italy.
Safety and efficacy of lentiviral hematopoietic stem cell Gene Therapy for Wiskott-Aldrich Syndrome
10:05 - 10:20 Maria Carmina Castiello - San Raffaele Telethon Institute for Gene Therapy, Italy.
Long-term efficacy of lentiviral vector-mediated gene therapy in restoring B-cell homeostasis in Wiskott-Aldrich syndrome patients
10:20 - 10:35 Michaela Semeraro - Centre d’Investigations Cliniques, INSERM CIC1419, Hôpital Necker Enfants malades - Université de Paris-Cité
Long-term Inflammatory manifestations post lentiviral hematopoietic stem/progenitor cell gene Therapy for Wiskott Aldrich syndrome
10:35 - 11:05 Gene Therapy Panel
Chair: Emma Morris
Participants : Alessandro Aiuti, Marina Cavazzana, Luigi Notarangelo, Suhag Parikh​
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“Lentiviral Gene Therapy for WAS, why is there variability in outcome and how we can address it?”
​11:05- 11:20 Break
​11:20- 11:25 Family Story
Gene Therapy Family Story - Priya Stephan (Short video)
​11:25- 11:55 Plenary session B
Alessia Cavazza - Infection, Immunity & Inflammation Department, UCL Institute of child health, London, UK.
"Development of a safe and efficient CRISPR/Cas gene editing platform for the treatment of Wiskott-Aldrich Syndrome"
11:55 - 12:30 Clinical International Session 1
Chair: Anders Fasth
11:55 - 12:05 Carmem Bonfim- Pediatric Transplant and Cellular Therapy Program - Duke University, NC, USA.
Haploidentical cell transplantation using post-transplantation cyclophosphamide for patients with Wiskott-Aldrich: Excellent outcomes and emerging challenges
12:05 - 12:15 Deepti Suri - Department of Pediatrics, Postgraduate Institute of Medical Education and Research, Chandigarh, India..
Correlation of Clinical Profiles, Immune Cells, Genotypes, and Regulatory Gene Expression in Indian Patients with XLT/WAS
12:15 - 12:25 Anahita Razaghian - IAARI and Hakim children hospital, Teheran, Iran. On line presentation
Clinical features and outcomes of 39 Wiskott-Aldrich syndrome patients from a single center of Iran
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12:25 - 12:30 Questions and answers and discussion
​12:30- 12:40 Family Story
Family Story - Emily Kinsel (Short video)
​12:40- 13:10 Plenary session C
Moderator: Luigi Notarangelo - Immune Deficiency Genetics Section, National Institute of Health, USA.
Participants: Alessandro Aiuti, Michael Albert, Fabio Candotti, Sumathi Iyengar, Emma Morris
“Evolving aspects of WAS: milder forms of the disease and carrier status – from disease perception to clinical implications”
​13:10- 14:10 Lunch
14:10 - 15:10 Clinical Session 2
Chair: Carmem Bonfim
14:10- 14:30 Emma Morris – Invited speaker, Director, UCL Division of Infection and Immunity, London, UK.
“Allogeneic HSCT for adults with WAS”
14:30 - 14:45 Tanja C. Vallée - Stem cell transplantation unit, Dr. von Haunersches University Children’s Hospital, Munich
Allogeneic HSCT with adjusted busulfan dosing results in excellent survival and cure rates for WAS
14:45 - 15:00 Yoji Sasahara – Dep. of Pediatrics & Pediatric Oncology, Tohoku University Graduate School of Medicine
Efficacy of rituximab for the treatment and prevention of autoimmunity in patients with WAS and XLT
15:00 - 15:10 - Questions and answers and discussion
15:10 - 15:45 Clinical Session 3
Chair: Suhag Parikh
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15:10 - 15:25 Maddalena Migliavacca - Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
New insights into Wiskott Aldrich Syndrome carriers: A multicentric observation
15:25 - 15:40 Markus Walz - Associate Partner, Magnolia Innovation, New Jersy, USA.
An International Study of Patient and Caregiver-Reported Burden and Quality of Life in Wiskott-Aldrich Syndrome
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15:40 - 15:45 - Questions & answers and discussion
15:45- 16:20 Clinical Session Panel
Chair: Michael Albert
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Participants : Participants: Alessandro Aiuti, Siobhan Burns, Fabio Candotti, Luigi Notarangelo
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“Towards a consensus statement in the management of WAS”
​16:20- 16:35 Break
​16:35 - 17:10 Research Session 1
Chair: Anna Villa
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16:35 - 16:50 Rhaissa Vieira - Department of Microbiology Tumor and Cell Biology, Karolinska Institutet, Sweden.
Therapeutic Candidate for Enhancing Platelet Production and Function in Wiskott-Aldrich Syndrome and X-Linked Thrombocytopenia
16:50 - 17:05 Lia Pinho - Department of Microbiology Tumor and Cell Biology, Karolinska Institutet, Sweden.
From Structure to Function: Understanding the impact of WASp Mutations on Actin Dynamics and T Cell Activation
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17:05 - 17:10 - Questions & answers and discussion
​17:10 - 18:20 Research Session 2
Chair: Alessandro Aiuti
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​17:10 - 17:30 Yatin Vyas – Invited speaker, Professor and Chair, Department of Pediatrics, Penn State Health Children's Hospital, USA.
Functional pairing of WASp and Fanconi anemia proteins in the cell nucleus- co-opting cosmic quantum entanglement
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17:30 - 17:45 Roberto Amadio - Cellular Immunology department, University of Trieste and ICGEB Trieste, Italy.
Actin polymerization by WASp limits nuclear envelope rupture and inflammation in macrophages
17:45 - 18:00 Jordan Chill - Department of Chemistry, Bar Ilan University, Israel.
Curation of a Structural WAS/XLT Database: Challenges and First Conclusions
18:00 - 18:15 Gianluca Baldanzi - Department of Translational Medicine, University of Piemonte Orientale, Novara, Italy.
DGKα is a negative regulator of megakaryopoiesis inhibited by WASp
18:15 - 18:20 Questions & answers and discussion answers and discussion
​18:20- 18:30 Summary
Anna Villa. Fabio Candotti, Sumathi Iyengar & Amir Kedar
​18:30- 19:15 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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