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用于雷特综合征MECP2脑修复的腺相关病毒载体新生儿脑室内递送方案。

Protocol for the neonatal intracerebroventricular delivery of adeno-associated viral vectors for brain restoration of MECP2 for Rett syndrome.

作者信息

Yang Kan, Li Tianshu, Geng Yixiao, Zhang Ru, Xu Zhankui, Wu Jun, Yuan Yiting, Zhang Yuefang, Qiu Zilong, Li Fei

机构信息

Department of Developmental and Behavioural Pediatric & Child Primary Care, Brain and Behavioural Research Unit of Shanghai Institute for Pediatric Research and MOE-Shanghai Key Laboratory for Children's Environmental Health of Xinhua Hospital & Songjiang Research Institute, Shanghai Jiao Tong University School of Medicine, Shanghai 200092, China; College of Materials and Chemical Engineering, Hunan Institute of Engineering, Xiangtan, Hunan 411104 , China.

Department of Developmental and Behavioural Pediatric & Child Primary Care, Brain and Behavioural Research Unit of Shanghai Institute for Pediatric Research and MOE-Shanghai Key Laboratory for Children's Environmental Health of Xinhua Hospital & Songjiang Research Institute, Shanghai Jiao Tong University School of Medicine, Shanghai 200092, China.

出版信息

STAR Protoc. 2024 Dec 20;5(4):103344. doi: 10.1016/j.xpro.2024.103344. Epub 2024 Sep 25.

DOI:10.1016/j.xpro.2024.103344
PMID:39331500
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11460457/
Abstract

Here, we present a protocol for neonatal intracerebroventricular (ICV) delivery of adeno-associated viral vectors (AAVs), achieving gene therapy for a Rett syndrome mouse model. We describe steps for preparing mouse lines, replacing foster mothers, sex typing, and genotyping. We then detail procedures for ICV delivery and validation through immunofluorescent and immunoblot techniques. This protocol is also applicable to preclinical gene therapy research that targets the neonatal mouse brain for other neurodevelopmental disorders. For complete details on the use and execution of this protocol, please refer to Yang et al..

摘要

在此，我们展示了一种用于向新生小鼠脑室内（ICV）递送腺相关病毒载体（AAV）的方案，该方案可实现对雷特综合征小鼠模型的基因治疗。我们描述了制备小鼠品系、更换代孕母鼠、性别鉴定和基因分型的步骤。然后，我们详细介绍了通过免疫荧光和免疫印迹技术进行脑室内递送及验证的程序。该方案也适用于针对新生小鼠脑进行其他神经发育障碍治疗的临床前基因治疗研究。有关该方案的使用和执行的完整详细信息，请参考Yang等人的研究。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e471/11460457/40945b650315/gr4.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e471/11460457/427448791ace/fx1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e471/11460457/048cc1d30fc4/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e471/11460457/c953f3f2a63f/gr2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e471/11460457/68486503cab1/gr3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e471/11460457/40945b650315/gr4.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e471/11460457/427448791ace/fx1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e471/11460457/048cc1d30fc4/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e471/11460457/c953f3f2a63f/gr2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e471/11460457/68486503cab1/gr3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e471/11460457/40945b650315/gr4.jpg

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本文引用的文献

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Mol Ther. 2023 Jul 5;31(7):2286-2295. doi: 10.1016/j.ymthe.2023.02.015. Epub 2023 Feb 18.
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In utero intracerebroventricular delivery of adeno-associated viral vectors to target mouse choroid plexus and cerebrospinal fluid.经子宫内侧脑室注射腺相关病毒载体靶向小鼠脉络丛和脑脊液。
STAR Protoc. 2023 Mar 17;4(1):101975. doi: 10.1016/j.xpro.2022.101975. Epub 2022 Dec 28.
3
Extension of the Lifespan of a Mouse Model of Rett Syndrome by Intracerebroventricular Delivery of MECP2.
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Neurosci Bull. 2023 Feb;39(2):297-302. doi: 10.1007/s12264-022-00974-y. Epub 2022 Nov 14.
4
Neural labeling and manipulation by neonatal intraventricular viral injection in mice.通过新生鼠脑室注射病毒进行神经标记和操作。
STAR Protoc. 2022 Jan 10;3(1):101081. doi: 10.1016/j.xpro.2021.101081. eCollection 2022 Mar 18.
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