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Katarina Kulhankova¹, Soumba Traore¹, Xue Cheng²

¹Department of Pediatrics, University of Iowa, Iowa City, IA, USA.

Nature communications

|December 5, 2023

概括

一个新的S315有效地将基因编辑工具传递给呼吸道细胞,显示出性纤维化 (CF) 治疗的前景. 这种基编辑器传递系统纠正CFTR突变,恢复肺功能.

科学领域:

基因治疗和基因编辑
呼吸系统药物呼吸系统药物
分子生物学分子生物学

背景情况:

对于囊性纤维化 (CF) 的基因编辑面临由于呼吸道上皮质障碍的挑战.
之前的研究表明,S10提供了与CRISPR相关的 (Cas) 核糖蛋白 (RNP) 用于气道细胞编辑.

研究的目的:

为基编辑器RNP开发一个改进的递送系统,以准气道表皮质.
评估基因编辑在呼吸道组织中的有效性和持久性.

主要方法:

衍生并测试了S315,用于传递基编辑器RNP.
在 rhesus macaques 中通过内气溶通过 Cy5 标记的 S315 .
与S315同时使用ABE8e-Cas9 RNP以向CCR5和CFTR R553X突变.
评估了编辑效率,小鼠的持久性和人类呼吸道表皮的功能恢复.

主要成果:

确认S315在的整个呼吸道传递.
在 rhesus 呼吸道表皮质中达到高达 5.3% 的编辑效率,针对 CCR5.5.
在小鼠中,经过编辑的上皮质的持续持续时间可达12个月.
通过纠正CFTR R553X突变,恢复了人类呼吸道上皮质中的离子通道功能.

结论:

S315是一种有效的输送载体,用于呼吸道中的基编辑器RNP.
这一策略显示了功能性纠正囊性纤维化中CFTR突变的治疗潜力.
这些发现支持进一步开发用于呼吸系统疾病的S315介导基因编辑.

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