Genetics and Rare Diseases Research Division, Bambino Gesù Children's Hospital, IRCCS, 00146 Rome, Italy.
Int J Mol Sci. 2021 Dec 20;22(24):13674. doi: 10.3390/ijms222413674.
To date, gene therapy has employed viral vectors to deliver therapeutic genes. However, recent progress in molecular and cell biology has revolutionized the field of stem cells and gene therapy. A few years ago, clinical trials started using stem cell replacement therapy, and the induced pluripotent stem cells (iPSCs) technology combined with CRISPR-Cas9 gene editing has launched a new era in gene therapy for the treatment of neurological disorders. Here, we summarize the latest findings in this research field and discuss their clinical applications, emphasizing the relevance of recent studies in the development of innovative stem cell and gene editing therapeutic approaches. Even though tumorigenicity and immunogenicity are existing hurdles, we report how recent progress has tackled them, making engineered stem cell transplantation therapy a realistic option.
迄今为止，基因治疗一直采用病毒载体来输送治疗基因。然而，分子和细胞生物学的最新进展彻底改变了干细胞和基因治疗领域。几年前，开始使用干细胞替代疗法进行临床试验，诱导多能干细胞（iPSC）技术与 CRISPR-Cas9 基因编辑的结合为神经障碍的基因治疗开启了一个新时代。在这里，我们总结了该研究领域的最新发现，并讨论了它们的临床应用，强调了最近在开发创新的干细胞和基因编辑治疗方法方面的研究相关性。尽管存在致瘤性和免疫原性等障碍，但我们报告了最近的进展如何解决这些问题，使得工程化干细胞移植治疗成为一种现实选择。
