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​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 VyasInvited 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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