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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Main Authors: 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.
Formato: Artigo
Idioma:Inglês
Publicado: 2010
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Article
Acceso en liña: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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