Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome

Wiskott-Aldrich syndrome (WAS) is an X-linked primary immunodeficiency with severe platelet abnormalities and complex immunodeficiency. Although clinical gene therapy approaches using lentiviral vectors have produced encouraging results, full immune and platelet reconstitution is not always achieved...

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Detalhes bibliográficos
Publicado no:Nat Commun
Main Authors: Rai, Rajeev, Romito, Marianna, Rivers, Elizabeth, Turchiano, Giandomenico, Blattner, Georges, Vetharoy, Winston, Ladon, Dariusz, Andrieux, Geoffroy, Zhang, Fang, Zinicola, Marta, Leon-Rico, Diego, Santilli, Giorgia, Thrasher, Adrian J., Cavazza, Alessia
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group UK 2020
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7423939/
https://ncbi.nlm.nih.gov/pubmed/32788576
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-020-17626-2
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