Lux Christopher T, Scharenberg Andrew M
Department of Pediatrics, Cancer and Blood Disorders Center, Seattle Children's Hospital, 4800 Sand Point Way NE, Seattle, WA 98105, USA.
Department of Pediatrics, Seattle Children's Hospital, 4800 Sand Point Way NE, Seattle, WA 98105, USA; Department of Immunology, Seattle Children's Hospital, 4800 Sand Point Way NE, Seattle, WA 98105, USA.
Hematol Oncol Clin North Am. 2017 Oct;31(5):787-795. doi: 10.1016/j.hoc.2017.05.002. Epub 2017 Jun 29.
Therapeutic gene editing is significant for medical advancement. Safety is intricately linked to the specificity of the editing tools used to cut at precise genomic targets. Improvements can be achieved by thoughtful design of nucleases and repair templates, analysis of off-target editing, and careful utilization of viral vectors. Advancements in DNA repair mechanisms and development of new generations of tools improve targeting of specific sequences while minimizing risks. It is important to plot a safe course for future clinical trials. This article reviews safety and specificity for therapeutic gene editing to spur dialogue and advancement.
治疗性基因编辑对医学进步具有重要意义。安全性与用于在精确基因组靶点进行切割的编辑工具的特异性密切相关。通过精心设计核酸酶和修复模板、分析脱靶编辑以及谨慎使用病毒载体，可以实现改进。DNA修复机制的进展和新一代工具的开发提高了对特定序列的靶向性，同时将风险降至最低。为未来的临床试验规划一条安全的道路很重要。本文综述了治疗性基因编辑的安全性和特异性，以促进对话和进步。
