Pomella Silvia, Rota Rossella
Department of Oncohematology, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
Front Chem. 2020 Mar 13;8:178. doi: 10.3389/fchem.2020.00178. eCollection 2020.
The RNA-guided clustered regularly interspaced palindromic repeats (CRISPR)/associated nuclease 9 (Cas9)-based genome editing technology has increasingly become a recognized method for translational research. In oncology, the ease and versatility of CRISPR/Cas9 has made it possible to obtain many results in the identification of new target genes and in unravel mechanisms of resistance to therapy. The majority of the studies have been made on adult tumors so far. In this mini review we present an overview on the major aspects of CRISPR/Cas9 technology with a focus on a group of rare pediatric malignancies, soft tissue sarcomas, on which this approach is having promising results.
基于RNA引导的成簇规律间隔回文重复序列（CRISPR）/相关核酸酶9（Cas9）的基因组编辑技术已日益成为一种公认的转化研究方法。在肿瘤学领域，CRISPR/Cas9的简便性和多功能性使得在鉴定新的靶基因以及揭示治疗抗性机制方面取得了许多成果。到目前为止，大多数研究都是针对成人肿瘤进行的。在这篇小型综述中，我们概述了CRISPR/Cas9技术的主要方面，重点关注一组罕见的儿科恶性肿瘤——软组织肉瘤，该方法在这方面正取得有前景的结果。
