Inefficient dystrophin expression after cord blood transplantation in DMD

INTRODUCTION: We report a boy who received two allogeneic stem cell transplantations from umbilical cord donors to treat chronic granulomatous disease (CGD). The CGD was cured after the second transplantation, but 2½ years later, he was diagnosed with Duchenne muscular dystrophy (DMD). METHODS: Exam...

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Hauptverfasser: Kang, Peter B., Lidov, Hart G.W., White, Alexander J., Mitchell, Matthew, Balasubramanian, Anuradha, Estrella, Elicia, Bennett, Richard R., Darras, Basil T., Shapiro, Frederic D., Bambach, Barbara J., Kurtzberg, Joanne, Gussoni, Emanuela, Kunkel, Louis M.
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2010
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2965738/
https://ncbi.nlm.nih.gov/pubmed/20513101
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/mus.21702
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