A Designer AAV Variant Permits Efficient Retrograde Access to Projection Neurons

效应器 神经科学 病毒载体 遗传增强 轴浆运输 基因传递 载体(分子生物学) 生物神经网络 投影(关系代数) 体内 计算机科学 生物 计算生物学 基因 细胞生物学 遗传学 重组DNA 算法
作者
D. Gowanlock R. Tervo,Bum-Yeol Hwang,Sarada Viswanathan,Thomas Gaj,Maria Lavzin,Kimberly Ritola,Sarah Lindo,Susan Michael,Е. П. Кулешова,David S. Ojala,Cheng-Chiu Huang,Charles R. Gerfen,Jackie Schiller,Joshua T. Dudman,Adam W. Hantman,Loren L. Looger,David V. Schaffer,Alla Y. Karpova
出处
期刊:Neuron [Cell Press]
卷期号:92 (2): 372-382 被引量:1136
标识
DOI:10.1016/j.neuron.2016.09.021
摘要

Efficient retrograde access to projection neurons for the delivery of sensors and effectors constitutes an important and enabling capability for neural circuit dissection. Such an approach would also be useful for gene therapy, including the treatment of neurodegenerative disorders characterized by pathological spread through functionally connected and highly distributed networks. Viral vectors, in particular, are powerful gene delivery vehicles for the nervous system, but all available tools suffer from inefficient retrograde transport or limited clinical potential. To address this need, we applied in vivo directed evolution to engineer potent retrograde functionality into the capsid of adeno-associated virus (AAV), a vector that has shown promise in neuroscience research and the clinic. A newly evolved variant, rAAV2-retro, permits robust retrograde access to projection neurons with efficiency comparable to classical synthetic retrograde tracers and enables sufficient sensor/effector expression for functional circuit interrogation and in vivo genome editing in targeted neuronal populations.Video AbstracteyJraWQiOiI4ZjUxYWNhY2IzYjhiNjNlNzFlYmIzYWFmYTU5NmZmYyIsImFsZyI6IlJTMjU2In0.eyJzdWIiOiI4YzNlNWNlMWIwMjEwNTMwMmU0NzU5Zjg1OGZhN2VjNSIsImtpZCI6IjhmNTFhY2FjYjNiOGI2M2U3MWViYjNhYWZhNTk2ZmZjIiwiZXhwIjoxNjc4OTIwNjUyfQ.PIawDOHpxANfHC02wRHgNR9xW5IWD2usKnoU6sy5jofdhbn59c1Wt7D4f3-Dl7r4FffS7kG5CmD0MAA4xhiDH4k_fpPFeDtnWqoPTdrhD2bVpfi4wTBnyie7NKaNMMjL-SOaDobd6kCRE9cYrdRXyy_uof_jf4gGsRH8RnPKrs1nbeaek5sg0XWmK1oC7db8iMRvW0ALbwNBLLAWZfnXfZsMX4AMI-aUIH1p7wjiFPGzO7nw_H7qJk6h1L6FJzADy3tveAdJWwrXNx7iceEi_V7JOsl98UpSp2H-4Z_-4Tw8PmXRAyPLcaDltdpiLVSvuFc_csSEUX62WnL3cf9iEA(mp4, (84.44 MB) Download video
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