5 results on '"Notarangelo, Luigi Daniele"'
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2. Successful Therapy of a Patient with a Novel STAT1 Gain of Function Mutation and Life-Threatening Cytopenias with Janus Kinase Inhibitor Ruxolitinib
3. Induced Pluripotent Stem Cells From a Patient with Reticular Dysgenesis Recapitulate Defective Myelopoiesis in-Vitro: A Disease Model to Enhance Our Understanding of a Rare Disease.
4. Somatic Gene Therapy for X-Linked Severe Combined Immunodeficiency Using a Self-Inactivating Modified Gammaretroviral Vector Results in An Improved Preclinical Safety Profile and Early Clinical Efficacy in a Human Patient
5. Different molecular behavior of CD40 mutants causing hyper-IgM syndrome
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