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Genome-edited adult stem cells: Next-generation advanced therapy medicinal products.
STEM CELLS Translational Medicine ( IF 5.4 ) Pub Date : 2020-03-06 , DOI: 10.1002/sctm.19-0338 Karim Benabdellah 1 , Sabina Sánchez-Hernández 1 , Araceli Aguilar-González 1, 2 , Noelia Maldonado-Pérez 1 , Alejandra Gutierrez-Guerrero 3 , Marina Cortijo-Gutierrez 1 , Iris Ramos-Hernández 1 , María Tristán-Manzano 1 , Pablo Galindo-Moreno 4 , Concha Herrera 5, 6 , Francisco Martin 1
STEM CELLS Translational Medicine ( IF 5.4 ) Pub Date : 2020-03-06 , DOI: 10.1002/sctm.19-0338 Karim Benabdellah 1 , Sabina Sánchez-Hernández 1 , Araceli Aguilar-González 1, 2 , Noelia Maldonado-Pérez 1 , Alejandra Gutierrez-Guerrero 3 , Marina Cortijo-Gutierrez 1 , Iris Ramos-Hernández 1 , María Tristán-Manzano 1 , Pablo Galindo-Moreno 4 , Concha Herrera 5, 6 , Francisco Martin 1
Affiliation
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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.
中文翻译：
基因组编辑的成人干细胞：下一代高级治疗药物。
在最近的几十年中，基因疗法已经实现了一场真正的革命，这种疗法已使多种不治之症得以治疗。细胞疗法在各种疾病的治疗中也取得了重大进展，特别是通过使用成体干细胞（ASC）。基因与细胞疗法（GCT）的结合为改善用于治疗多种疾病的先进治疗药物开辟了新的机遇。尽管GCT具有巨大潜力，但逆转录病毒载体的使用在致癌基因反式激活和缺乏生理表达方面有很大的局限性。最近，基因治疗师已将基因编辑（GE）技术作为替代策略。在本文中，我们讨论了使用GE技术改进基于ASC的GCT方法的潜在好处。我们将首先简要介绍不同的GE平台和技术，然后重点介绍已成功用于治疗动物模型疾病的关键治疗方法。最后，我们讨论了ASC GE是否可以在GCT环境中真正替代逆转录病毒载体。
更新日期：2020-03-06
中文翻译：
基因组编辑的成人干细胞：下一代高级治疗药物。
在最近的几十年中，基因疗法已经实现了一场真正的革命，这种疗法已使多种不治之症得以治疗。细胞疗法在各种疾病的治疗中也取得了重大进展，特别是通过使用成体干细胞（ASC）。基因与细胞疗法（GCT）的结合为改善用于治疗多种疾病的先进治疗药物开辟了新的机遇。尽管GCT具有巨大潜力，但逆转录病毒载体的使用在致癌基因反式激活和缺乏生理表达方面有很大的局限性。最近，基因治疗师已将基因编辑（GE）技术作为替代策略。在本文中，我们讨论了使用GE技术改进基于ASC的GCT方法的潜在好处。我们将首先简要介绍不同的GE平台和技术，然后重点介绍已成功用于治疗动物模型疾病的关键治疗方法。最后，我们讨论了ASC GE是否可以在GCT环境中真正替代逆转录病毒载体。
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