CLINICAL TRIAL OF HEMATOPOIETIC STEM CELL GENE THERAPY FOR WISKOTT-ALDRICH-SYNDROME
- 8 Anni 2010/2018
- 4.079.127€ Totale Fondi
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Pubblicazioni Scientifiche
- 2014-01-01 CURRENT GENE THERAPY
Gene Therapy for Wiskott-Aldrich Syndrome
- 2016-12-01 CYTOTHERAPY
Bone marrow-derived CD34- fraction: A rich source of mesenchymal stromal cells for clinical application
- 2020-11-24 FRONTIERS IN IMMUNOLOGY
Mild SARS-CoV-2 Infection After Gene Therapy in a Child With Wiskott-Aldrich Syndrome: A Case Report
- 2020-01-24 FRONTIERS IN PEDIATRICS
Hematopoietic Stem Cell Transplantation for Combined Immunodeficiencies, on Behalf of IEWP-EBMT
- 2016-08-01 JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
A novel genomic inversion in Wiskott-Aldrich-associated autoinflammation
- 2018-03-07 MOLECULAR THERAPY
Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety
- 2018-04-06 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