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

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

A platform for systemic therapeutic transgene expression independent of patient mutations needs a safe and highly transcribed locus. Here the authors ex vivo edit HPSCs using CRISPR-Cas9 to integrate transgenes under the α-globin promoter to achieve erythroid specific expression.

Guardado en:
Detalles Bibliográficos
Autores principales: Giulia Pavani, Marine Laurent, Anna Fabiano, Erika Cantelli, Aboud Sakkal, Guillaume Corre, Peter J. Lenting, Jean-Paul Concordet, Magali Toueille, Annarita Miccio, Mario Amendola
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2020
Materias:
Science
Q
Acceso en línea:https://doaj.org/article/bd6f18164a234100805135163a84adaa
Etiquetas: Agregar Etiqueta
Sin Etiquetas, Sea el primero en etiquetar este registro!

Ejemplares similares

Ejemplares similares