Abstract：
Engineering and reprogramming cells has significant therapeutic potential to treat a wide range of diseases, by replacing missing or defective proteins, to provide transcription factors (TFs) to reprogram cell phenotypes, or to provide enzymes such as RNA-guided Cas9 derivatives for CRISPR-based cell engineering. While viral vector-mediated gene transfer has played an important role in this field, the use of mRNA avoids safety concerns associated with the integration of DNA into the host cell genome, making mRNA particularly attractive for in vivo applications. Widespread application of mRNA for cell engineering is limited by its instability in the biological environment and challenges involved in the delivery of mRNA to its target site. In this review, we examine challenges that must be overcome to develop effective therapeutics.
摘要：
改造和重编程细胞在治疗多种疾病方面具有巨大的治疗潜力，通过替换缺失或有缺陷的蛋白质，提供转录因子（TFs）来重新编程细胞表型，或提供用于基于CRISPR的细胞工程的RNA指导的Cas9衍生物等酶。而病毒载体介导的基因转移在这一领域发挥了重要作用,mRNA的使用避免了与DNA整合到宿主细胞基因组相关的安全问题，使mRNA对体内应用特别有吸引力。mRNA在细胞工程中的广泛应用受到其在生物环境中的不稳定性和mRNA递送至其靶位点所涉及的挑战的限制。在这次审查中,我们研究开发有效疗法必须克服的挑战。
