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新型AAV8基因疗法NGGT006用于纯合子家族性高胆固醇血症,在3周内将LDL-C降低97.5%。效果持续52周。了解谁有资格接受治疗。
NEJM发表了基因疗法VERVE-102的1b期结果:单次注射后LDL降低62%。分析其疗效、风险及对高胆固醇血症患者的前景。了解更多。