Intellia治疗疗法的CRISPR疗法在治疗ATTR 氨基虫病方面显示出有希望的结果。 Intellia Therapeutics' CRISPR therapy shows promising results in treating ATTR amyloidosis.

Intellia治疗学报告称,对Nexiguran ziclumeran的第一阶段试验取得了积极的成果,该试验是以CRISPR为基础的对ATTR 氨基质病的基因治疗。 Intellia Therapeutics reported positive results from a Phase 1 trial of nexiguran ziclumeran, a CRISPR-based gene therapy for ATTR amyloidosis. 治疗显示,疾病标记迅速和持久地减少,单剂量后患者状况得到改善。 The treatment showed rapid and lasting reductions in disease markers and improvements in patient conditions after a single dose. 发现这种治疗是安全的和良好的,在这种疾病的心脏和神经相关症状中都观察到了好处。 The therapy was found to be safe and well-tolerated, with benefits observed in both heart and nerve-related symptoms of the disease.