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Reduced toxicity conditioning and a high CD34(+) cell dose can achieve full donor chimerism in DOCK8 deficiency
BACKGROUND: Biallelic mutations in the dedicator of cytokinesis 8 (DOCK8) gene were identified as the cause of combined immunodeficiency in 2009. Survival rates without hematopoietic stem cell transplant in patients with DOCK8 deficiency decline from 87% at 10 years to 33% at 30 years. Hematopoietic...
Autores principales: | Pandrowala, Ambreen, Sharma, Ajay Narayan, Kakunje, Manasa, Bodhanwala, Minnie, Hiwarkar, Prashant |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10510004/ https://www.ncbi.nlm.nih.gov/pubmed/37779528 http://dx.doi.org/10.1016/j.jacig.2023.100106 |
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