@misc{10481/62706,
year = {2020},
url = {http://hdl.handle.net/10481/62706},
abstract = {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.},
organization = {European Regional Development Fund
(FEDER), Grant/Award Numbers: PI18/01610,
PI18/00330, PI18/00337, grants PI12/01097;
Spanish ISCIII Health Research Fund},
publisher = {WILEY},
keywords = {Adult stem cells},
keywords = {CRISPR},
keywords = {Electroporation},
keywords = {Gene delivery systems in vivo or in vitro},
keywords = {Gene therapy},
keywords = {Hematopoietic stem cells (HSCs)},
keywords = {Mesenchymal stem cells (MSCs)},
keywords = {Pluripotent hemopoietic stem cells},
title = {Genome-edited adult stem cells: Next-generation advanced therapy medicinal products},
doi = {10.1002/sctm.19-0338},
author = {Benabdellah, Karim and Sánchez Hernández, Sabina and Aguilar González, Araceli and Maldonado Pérez, Noelia and Cortijo Gutierrez, Marina and Ramos Hernández, Iris and Tristán Manzano, María and Martín, Francisco},
}