As Parkinson's disease progresses, levodopa treatment loses efficacy, partly through the loss of the endogenous dopamine‐synthesizing enzyme L‐amino acid decarboxylase (AADC). In the phase I PD‐1101 study, putaminal administration of VY‐AADC01, an investigational adeno‐associated virus serotype‐2 vector for delivery of the AADC gene in patients with advanced Parkinson's disease, was well tolerated, improved motor function, and reduced antiparkinsonian medication requirements.

中文翻译：

---

芳香族 L-氨基酸脱羧酶基因疗法可增强帕金森病的左旋多巴反应

随着帕金森病的进展，左旋多巴治疗失去疗效，部分原因是内源性多巴胺合成酶 L-氨基酸脱羧酶 (AADC) 的丧失。在 I 期 PD-1101 研究中，VY-AADC01（一种用于在晚期帕金森病患者中传递 AADC 基因的研究性腺相关病毒血清型 2 载体）的壳核给药具有良好的耐受性，改善了运动功能，并减少了抗帕金森病用药要求。