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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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Detalles Bibliográficos
Autores principales:	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:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2020
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7391635/ https://www.ncbi.nlm.nih.gov/pubmed/32728076 http://dx.doi.org/10.1038/s41467-020-17552-3

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