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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 - TBA
​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 - 11:05 Research Session 1 
Chair: Lisa Westerberg

​09:50 - 10:10 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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10:10 - 10:25 Roberto Amadio - Cellular Immunology department, University of Trieste and ICGEB Trieste, Italy.

Actin polymerization by WASp limits nuclear envelope rupture and inflammation in macrophages

 

10:25 - 10:40 Jordan Chill - Department of Chemistry, Bar Ilan University, Israel.  

Curation of a Structural WAS/XLT Database: Challenges and First Conclusions

 

10:40 - 10:55 Gianluca Baldanzi - Department of Translational Medicine, University of Piemonte Orientale, Novara, Italy.   

DGKα is a negative regulator of megakaryopoiesis inhibited by WASp

 

10:55 - 11:05 Questions & answers and discussion  answers and discussion  

​11:05- 11:20 Break
​11:20- 11:50 Plenary session B
Luigi Notarangelo - Immune Deficiency Genetics Section, National Institute of Health, USA.
'Wiskott-Aldrich syndrome – Where do we stand? Where should we go?'
​11:50 - 12:30 Research Session 2 

Chair: Anna Villa

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11:50 - 12:05  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

 

12:05 - 12:20  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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12:20 - 12:30  - Questions & answers and discussion  

12:30 - 13:00 Clinical International Session 1

Chair: Anders Fasth

 

12:30 - 12:40  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

 

12:40 - 12:50  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       

 

12:50 - 13:00  Anahita Razaghian - IAARI and Hakim children hospital, Teheran, Iran.

Clinical features and outcomes of 39 Wiskott-Aldrich syndrome patients from a single center of Iran

​13:00- 13:30 Plenary session C
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'

​13:30- 14:30 Lunch 
14:30 - 15:15 Gene Therapy Session

Chair: Fabio Candotti

14:30 - 14:45  Francesca Ferrua - San Raffaele Telethon Institute for Gene Therapy, Italy.

Safety and efficacy of lentiviral hematopoietic stem cell Gene Therapy for Wiskott-Aldrich Syndrome

 

14:45 - 15:00  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

 

15:00 - 15:15  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

 

15:15 - 15:45 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?”

15:45 - 16:45 Clinical Session 2

Chair: Carmem Bonfim

 

15:45 - 16:05 Emma Morris – Invited speaker, Director, UCL Division of Infection and Immunity, London, UK.

 “Allogeneic HSCT for adults with WAS”

 

16:05 - 16:20 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

 

16:20 - 16:35 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

 

16:35 - 16:45 - Questions and answers and discussion

​16:45- 16:55 Family Story 
Young Adult Family Story - TBA
​16:55- 17:15 Break
17:15 - 17:50 Clinical Session 3

Chair: Alessandro Aiuti

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17:15 - 17:30  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

 

17:30 - 17:45  Alexandra Wilds - 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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17:45 - 17:50   - Questions & answers and discussion  

17:50- 18:20 Clinical Session Panel

Chair: Michael Albert

Participants : TBA 

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“Towards a consensus statement in the management of WAS” 

​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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