CRISPR早期试验显示一次性基因编辑可以大幅降低胆固醇,但治疗仍然在试验中。
Early CRISPR trials show one-time gene edits can drastically lower cholesterol, but treatments remain experimental.
科学家们正在利用CRISPR测试基因编辑疗法,以便在有几十人参加的早期试验中试验永久降低高胆固醇这一主要心脏病风险因素。
Scientists are testing gene-editing therapies using CRISPR to permanently lower high cholesterol, a major heart disease risk factor, in early-stage trials with a few dozen participants.
两家公司,包括一家由Eli Lilly支持的公司,正在针对肝脏中的ANGPTL3和PCSK9等基因,一次治疗显示LDL胆固醇和三甘酸酯的半衰期长达数月至数年。
Two companies, including one backed by Eli Lilly, are targeting genes like ANGPTL3 and PCSK9 in the liver, with a single treatment showing up to 50% reductions in LDL cholesterol and triglycerides, lasting months to years.
虽然结果大有希望,而且有一天可以提供一次性解决办法,但治疗方法仍然是试验性的,具有长期安全性、非目标影响以及肝炎等风险仍不得而知。
While results are promising and could one day offer a one-time fix, the treatments remain experimental, with long-term safety, off-target effects, and risks like liver inflammation still unknown.
专家强调,他汀类药物和生活方式改变仍是标准治疗,广泛使用还需数年时间。
Experts stress that statins and lifestyle changes remain the standard of care, and widespread use is years away.