About Wiskott-Aldrich Syndrome
Wiskott-Aldrich Syndrome is an X-linked disorder caused by mutations in the WAS gene encoding WASp, a cytoskeletal regulatory protein expressed exclusively in hematopoietic cells, leading to the classic triad of microthrombocytopenia, eczema, and combined immunodeficiency. Immune dysfunction affects T cells, B cells, and NK cells, resulting in susceptibility to bacterial, viral, and opportunistic infections along with autoimmune complications and an elevated risk of lymphoma. Gene therapy approaches using lentiviral vectors have demonstrated curative potential in trials for patients lacking a suitable transplant donor.
Common Clinical Features
Clinical Trial Eligibility Tips
What to know before applying to Wiskott-Aldrich Syndrome trials.
Gene therapy trials typically require confirmed pathogenic WAS mutation and absence of matched sibling donor; HLA typing of all family members should be completed before applying
Platelet count thresholds (often >10,000/µL without support) and absence of active bleeding are common safety eligibility criteria
Autoimmune disease activity score and current immunosuppression regimen must be documented; active severe autoimmunity may exclude participation
Patient Resources
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