Low-Dose Intracerebroventricular Delivery of a Second-Generation AAV Gene Therapy for Spinal Muscular Atrophy Achieves Efficient and Toxicity-Free Motor Function Rescue in Mice

Xiupeng Chen,Qing Xie, Hong Ma, Yijie Ma, Leila Jalinous, Jun Yang, Yunxiang Zhu,Qin Su,Phillip W. L. Tai,Guangping Gao,Jun Xie

MOLECULAR THERAPY(2023)

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