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Busulfan Pharmacokinetics in ADA SCID Gene Therapy
The pharmacokinetics of low dose busulfan (BU) were investigated as a non-myeloablative conditioning regimen for autologous gene therapy (GT) in pediatric subjects with adenosine deaminase-deficient severe combined immunodeficiency (ADA SCID). In three successive clinical trials, which included eith...
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| Publicado en: | Biol Blood Marrow Transplant |
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| Main Authors: | , , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado: |
2020
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| Assuntos: | |
| Acceso en liña: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7529956/ https://ncbi.nlm.nih.gov/pubmed/32653625 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.bbmt.2020.07.004 |
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