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Hematopoietic Cell Transplantation for Adenosine Deaminase Severe Combined Immunodeficiency—Improved Outcomes in the Modern Era

Current treatment for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID) includes enzyme replacement therapy (ERT), allogeneic hematopoietic stem cell transplant (HSCT), or ex vivo corrected autologous hematopoietic stem cell gene therapy. Historic data show HSCT survival is...

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Detalles Bibliográficos
Autores principales: Ghimenton, Elisabetta, Flinn, Aisling, Lum, Su Han, Leahy, Timothy R., Nademi, Zohreh, Owens, Stephen, Williams, Eleri, Flood, Terrence, Hambleton, Sophie, Slatter, Mary, Gennery, Andrew R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer US 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9166891/
https://www.ncbi.nlm.nih.gov/pubmed/35288820
http://dx.doi.org/10.1007/s10875-022-01238-0