11 results on '"Rivella, Stefano"'
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2. Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiency
3. Combination of a TGF‐β ligand trap (RAP‐GRL) and TMPRSS6‐ASO is superior for correcting β‐thalassemia
4. Effective Gene Therapy for Metachromatic Leukodystrophy Achieved with Minimal Lentiviral Genomic Integrations
5. Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of Multiple Sulfatase Deficiency
6. Optimizing lentiviral genomic integrations to cure beta-thalassemia: The least required for success?
7. Single vs. dual transgene gene therapy for multiple sulfatase deficiency
8. Iron restriction in sickle cell disease: When less is more.
9. Single vs. dual transgene ex vivo gene therapy for multiple sulfatase deficiency
10. Effective Gene Therapy for Metachromatic Leukodystrophy Achieved with Minimal Lentiviral Genomic Integrations.
11. Optimizing lentiviral genomic integrations to cure beta-thalassemia: The least required for success?
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