Genome-edited adult stem cells: Next-generation advanced therapy medicinal products Benabdellah, Karim Sánchez Hernández, Sabina Aguilar González, Araceli Maldonado Pérez, Noelia Cortijo Gutierrez, Marina Ramos Hernández, Iris Tristán Manzano, María Martín, Francisco Adult stem cells CRISPR Electroporation Gene delivery systems in vivo or in vitro Gene therapy Hematopoietic stem cells (HSCs) Mesenchymal stem cells (MSCs) Pluripotent hemopoietic stem cells Over recent decades, gene therapy, which has enabled the treatment of several incurable diseases, has undergone a veritable revolution. Cell therapy has also seen major advances in the treatment of various diseases, particularly through the use of adult stem cells (ASCs). The combination of gene and cell therapy (GCT) has opened up new opportunities to improve advanced therapy medicinal products for the treatment of several diseases. Despite the considerable potential of GCT, the use of retroviral vectors has major limitations with regard to oncogene transactivation and the lack of physiological expression. Recently, gene therapists have focused on genome editing (GE) technologies as an alternative strategy. In this review, we discuss the potential benefits of using GE technologies to improve GCT approaches based on ASCs. We will begin with a brief summary of different GE platforms and techniques and will then focus on key therapeutic approaches that have been successfully used to treat diseases in animal models. Finally, we discuss whether ASC GE could become a real alternative to retroviral vectors in a GCT setting. 2020-06-24T11:49:03Z 2020-06-24T11:49:03Z 2020 journal article Benabdellah, K., Sánchez‐Hernández, S., Aguilar‐González, A., Maldonado‐Pérez, N., Gutierrez‐Guerrero, A., Cortijo‐Gutierrez, M., ... & Martin, F. (2020). Genome‐edited adult stem cells: Next‐generation advanced therapy medicinal products. Stem Cells Translational Medicine, 9(6), 674-685. [Doi: 10.1002/sctm.19-0338] http://hdl.handle.net/10481/62706 10.1002/sctm.19-0338 eng http://creativecommons.org/licenses/by-nc-nd/3.0/es/ open access Atribución-NoComercial-SinDerivadas 3.0 España WILEY