Flotte et al. describe the first test of adeno-associated virus-based gene therapy for Tay-Sachs disease in humans. Delivery to the thalamus and cerebrospinal fluid was found to be broadly safe, providing a firm basis for future clinical trials.

中文翻译：

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迈向 Tay-Sachs 病的基因治疗

弗洛特等人。描述了针对人类 Tay-Sachs 病的基于腺相关病毒的基因治疗的首次测试。发现输送到丘脑和脑脊液是广泛安全的，为未来的临床试验提供了坚实的基础。