Correction of β-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients

β-Thalassemia is a common monogenic disorder due to mutations in the β-globin gene and gene therapy, based on autologous transplantation of genetically corrected haematopoietic stem cells (HSCs), holds the promise to treat patients lacking a compatible bone marrow (BM) donor. We recently showed corr...

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Detalhes bibliográficos
Main Authors: Roselli, Emanuela Anna, Mezzadra, Riccardo, Frittoli, Marta Claudia, Maruggi, Giulietta, Biral, Erika, Mavilio, Fulvio, Mastropietro, Fabrizio, Amato, Antonio, Tonon, Giovanni, Refaldi, Chiara, Cappellini, Maria Domenica, Andreani, Marco, Lucarelli, Guido, Roncarolo, Maria Grazia, Marktel, Sarah, Ferrari, Giuliana
Formato: Artigo
Idioma:Inglês
Publicado em: WILEY-VCH Verlag 2010
Assuntos:
Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3377331/
https://ncbi.nlm.nih.gov/pubmed/20665635
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/emmm.201000083
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