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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: WILEY-VCH Verlag 2010
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3377331/
https://www.ncbi.nlm.nih.gov/pubmed/20665635
http://dx.doi.org/10.1002/emmm.201000083

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3377331/
https://www.ncbi.nlm.nih.gov/pubmed/20665635
http://dx.doi.org/10.1002/emmm.201000083

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