Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins
oleh: Giulia Pavani, Marine Laurent, Anna Fabiano, Erika Cantelli, Aboud Sakkal, Guillaume Corre, Peter J. Lenting, Jean-Paul Concordet, Magali Toueille, Annarita Miccio, Mario Amendola
| Format: | Article |
|---|---|
| Diterbitkan: | Nature Portfolio 2020-07-01 |
Deskripsi
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.