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

Internet

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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