在生命科学领域，每一次基因编辑技术的革新都可能深刻影响到未来的疾病治疗。近年来，基因编辑的迅猛发展使得RNA编辑技术以其独特的优势引起了广泛关注。与传统的DNA编辑手段相比，RNA编辑在安全性上具备了明显的优势，它不仅可控而且是可逆的，这为治疗众多遗传性疾病提供了新的可能性。然而，现有的RNA编辑技术大多数集中在单碱基的编辑上，这无疑限制了其在实际应用中的广泛性和有效性。

站在技术代际更替的转折点，三个趋势已清晰可见：RNA编辑将逐步从单碱基修补转向结构重塑，治疗窗口期从胚胎阶段延展至全生命周期，基因药物开发范式从'纠正错误'转向'功能重建'。对于产业界而言，当务之急是建立RNA结构数据库以优化导向设计，开发体内递送系统突破肝脏靶向限制，同时构建动态监测体系防范脱靶风险。这场由中国人引领的基因编辑革命，正在重新定义生物医药创新的全球版图。 返回搜狐，查看更多 ...

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By Balambal Suryanarayanan CRISPR-Cas9 is a repurposed gene editing tool that allows scientists to cut, replace, and insert pieces of DNA in precise regions along the strand. Courtesy of NIH Image ...

The CRISPR-Cas gene scissors offer a wide range of potential applications, from the treatment of genetic diseases to antiviral therapies and diagnostics. However, to safely harness their powers, ...