Boucher Paul, Cui Xiaoxia, Curiel David T
Department of Biomedical Engineering, McKelvey School of Engineering, Washington University in Saint Louis, St. Louis, MO 63130, USA; Division of Cancer Biology, Department of Radiation Oncology, School of Medicine, Washington University in Saint Louis, St. Louis, MO 63110, USA.
Genome Engineering & iPSC Center, Department of Genetics, School of Medicine, Washington University in Saint Louis, St. Louis, MO 63110, USA.
J Control Release. 2020 Nov 10;327:788-800. doi: 10.1016/j.jconrel.2020.09.003. Epub 2020 Sep 3.
Harnessing the bacterial clustered regularly interspaced short palindromic repeats (CRISPR) system for genome editing in eukaryotes has revolutionized basic biomedical research and translational sciences. The ability to create targeted alterations of the genome through this easy to design system has presented unprecedented opportunities to treat inherited disorders and other diseases such as cancer through gene therapy. A major hurdle is the lack of an efficient and safe in vivo delivery system, limiting most of the current gene therapy efforts to ex vivo editing of extracted cells. Here we discuss the unique features of adenoviral vectors that enable tissue specific and efficient delivery of the CRISPR-Cas machinery for in vivo genome editing.
利用细菌的成簇规律间隔短回文重复序列（CRISPR）系统在真核生物中进行基因组编辑，彻底改变了基础生物医学研究和转化科学。通过这个易于设计的系统对基因组进行靶向改造的能力，为通过基因疗法治疗遗传性疾病和其他疾病（如癌症）带来了前所未有的机遇。一个主要障碍是缺乏高效且安全的体内递送系统，这使得目前大多数基因治疗工作仅限于对提取细胞进行离体编辑。在此，我们讨论腺病毒载体的独特特性，这些特性能够实现CRISPR-Cas机制的组织特异性和高效递送，用于体内基因组编辑。
