Giono Luciana E
Instituto de Fisiología, Biología Molecular y Neurociencias (IFIBYNE), CONICET-Universidad de Buenos Aires, Buenos Aires, Argentina. E-mail:
Medicina (B Aires). 2017;77(5):405-409.
The development of techniques that allow the precise and efficient edition of the genome of living cells is one of the main goals of biomedical research. Over the last few decades, a number of genome editing tools have been developed, the most prominent being the CRISPR/Cas9 system, a bacterial defense mechanism that has been redesigned for its use in other cellular systems. The accessibility, both technical and economical, and the enormous potential of CRISPR/Cas9 have contributed to an almost unprecedented revolution in the biomedical sciences and represent an important step forward in the field of gene therapy that needs, however, to be taken cautiously.
开发能够精确且高效地编辑活细胞基因组的技术是生物医学研究的主要目标之一。在过去几十年里，已经开发出了多种基因组编辑工具，其中最突出的是CRISPR/Cas9系统，这是一种细菌防御机制，已被重新设计用于其他细胞系统。CRISPR/Cas9在技术和经济方面的可及性以及巨大潜力，促成了生物医学科学领域几乎史无前例的变革，代表了基因治疗领域向前迈出的重要一步，然而，这一步需要谨慎对待。
