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


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 impr

SUBMITTER: Benabdellah K 

PROVIDER: S-EPMC7214650 | biostudies-literature | 2020 Jun

REPOSITORIES: biostudies-literature