Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins

Targeted genome editing has a great therapeutic potential to treat disorders that require protein replacement therapy. To develop a platform independent of specific patient mutations, therapeutic transgenes can be inserted in a safe and highly transcribed locus to maximize protein expression. Here,...

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Publicado en:Nat Commun
Main Authors: Pavani, Giulia, Laurent, Marine, Fabiano, Anna, Cantelli, Erika, Sakkal, Aboud, Corre, Guillaume, Lenting, Peter J., Concordet, Jean-Paul, Toueille, Magali, Miccio, Annarita, Amendola, Mario
Formato: Artigo
Idioma:Inglês
Publicado: Nature Publishing Group UK 2020
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC7391635/
https://ncbi.nlm.nih.gov/pubmed/32728076
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-020-17552-3
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