Biblio
Musculoskeletal manifestations in mucopolysaccharidosis type I (Hurler syndrome) following hematopoietic stem cell transplantation. Orphanet J Rare Dis. 2016;11(1):93.
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Favorable outcomes of hematopoietic stem cell transplantation in children and adolescents with Diamond-Blackfan anemia. Blood Adv. 2020;4(8):1760-1769.
HSCT is effective in patients with PSTPIP1-associated myeloid-related proteinemia inflammatory (PAMI) syndrome. J Allergy Clin Immunol. 2020.
Influence of anti-thymocyte globulin plasma levels on outcome parameters in stem cell transplanted children. Int Immunopharmacol. 2020;83:106371.
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Finding a balance in reduced toxicity hematopoietic stem cell transplantation for thalassemia: role of infused CD3+ cell count and immunosuppression. Bone Marrow Transplant. 2024.
Precise CRISPR-Cas9 gene repair in autologous memory T cells to treat familial hemophagocytic lymphohistiocytosis. Sci Immunol. 2024;9(92):eadi0042.