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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Veröffentlicht in:Biol Blood Marrow Transplant
Hauptverfasser: Bradford, Kathryn L., Liu, Siyu, Krajinovic, Maja, Ansari, Marc, Garabedian, Elizabeth, Tse, John, Wang, Xiaoyan, Shaw, Kit L., Gaspar, H. Bobby, Candotti, Fabio, Kohn, Donald B.
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2020
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Article
Online Zugang: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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