RT Journal Article
T1 Genome-edited adult stem cells: Next-generation advanced therapy medicinal products.
A1 Benabdellah, Karim
A1 Sanchez-Hernandez, Sabina
A1 Aguilar-Gonzalez, Araceli
A1 Maldonado-Perez, Noelia
A1 Gutierrez-Guerrero, Alejandra
A1 Cortijo-Gutierrez, Marina
A1 Ramos-Hernandez, Iris
A1 Tristan-Manzano, Maria
A1 Galindo-Moreno, Pablo
A1 Herrera, Concha
A1 Martin, Francisco
K1 CRISPR
K1 Adult stem cells
K1 Electroporation
K1 Gene delivery systems in vivo or in vitro
K1 Gene therapy
K1 Hematopoietic stem cells (HSCs)
K1 Mesenchymal stem cells (MSCs)
K1 Pluripotent hemopoietic stem cells
AB 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.
PB Oxford University Press
YR 2020
FD 2020-01-10
LK http://hdl.handle.net/10668/15208
UL http://hdl.handle.net/10668/15208
LA en
NO Benabdellah K, Sánchez-Hernández S, Aguilar-González A, Maldonado-Pérez N, Gutierrez-Guerrero A, Cortijo-Gutierrez M, et al. Genome-edited adult stem cells: Next-generation advanced therapy medicinal products. Stem Cells Transl Med. 2020 Jun;9(6):674-685
DS RISalud
RD Apr 7, 2025