Genome-edited adult stem cells: Next-generation advanced therapy medicinal products.

Stem Cells Translational Medicine
Karim BenabdellahFrancisco Martin

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.

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Methods Mentioned

BETA
genetic modification
gene modification
genetic modifications

Clinical Trials Mentioned

NCT03164135
NCT02500849
NCT03207009
NCT03745287
NCT03432364
NCT03728322
NCT03653247
NCT02931071

Software Mentioned

ASC

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