Victoria Bordon

Prevalence, clinical characteristics, and prognosis of GATA2-related myelodysplastic syndromes in children and adolescents

Marcin W. Włodarski, Shinsuke Hirabayashi, Víctor Manuel López Pastor et al. · Blood · 2015 · 380 citations · Full text

Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study

Mieke Aldenhoven, Robert Wynn, Paul J. Orchard et al. · Blood · 2015 · 309 citations · Full text

Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study

Federica Barzaghi, Laura Amaya, Bénédicte Neven et al. · Journal of Allergy and Clinical Immunology · 2017 · 301 citations · Full text

Hematopoietic Stem Cell Gene Therapy for Adenosine Deaminase–Deficient Severe Combined Immunodeficiency Leads to Long-Term Immunological Recovery and Metabolic Correction

H. Bobby Gaspar, Samantha Cooray, Kimberly Gilmour et al. · Science Translational Medicine · 2011 · 281 citations · Full text

Mutations in TRNT1 cause congenital sideroblastic anemia with immunodeficiency, fevers, and developmental delay (SIFD)

Pranesh Chakraborty, Klaus Schmitz‐Abe, Erin K. Kennedy et al. · Blood · 2014 · 176 citations · Full text