CLINICAL TRIAL OF HEMATOPOIETIC STEM CELL GENE THERAPY FOR WISKOTT-ALDRICH-SYNDROME

Pubblicazioni Scientifiche

• CURRENT GENE THERAPY

  Gene Therapy for Wiskott-Aldrich Syndrome

• CYTOTHERAPY

  Bone marrow-derived CD34- fraction: A rich source of mesenchymal stromal cells for clinical application

• FRONTIERS IN IMMUNOLOGY

  Mild SARS-CoV-2 Infection After Gene Therapy in a Child With Wiskott-Aldrich Syndrome: A Case Report

• FRONTIERS IN PEDIATRICS

  Hematopoietic Stem Cell Transplantation for Combined Immunodeficiencies, on Behalf of IEWP-EBMT

• JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

  A novel genomic inversion in Wiskott-Aldrich-associated autoinflammation

• MOLECULAR THERAPY

  Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety

• ORPHANET JOURNAL OF RARE DISEASES

  Gene therapy in rare diseases: the benefits and challenges of developing a patient-centric registry for Strimvelis in ADA-SCID