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

Autor:	Yang K; 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. Electronic address: kanyang@shsmu.edu.cn., Li T; 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., Geng Y; 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., Zhang R; College of Materials and Chemical Engineering, Hunan Institute of Engineering, Xiangtan, Hunan 411104 , China., Xu Z; College of Materials and Chemical Engineering, Hunan Institute of Engineering, Xiangtan, Hunan 411104 , China., Wu J; 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., Yuan Y; 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., Zhang Y; 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., Qiu Z; 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. Electronic address: qiuzilong@shsmu.edu.cn., Li F; 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. Electronic address: feili@shsmu.edu.cn.
Jazyk:	angličtina
Zdroj:	STAR protocols [STAR Protoc] 2024 Dec 20; Vol. 5 (4), pp. 103344. Date of Electronic Publication: 2024 Sep 25.
DOI:	10.1016/j.xpro.2024.103344
Abstrakt:	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. 1 . Competing Interests: Declaration of interests The authors declare no competing interests. (Copyright © 2024 The Author(s). Published by Elsevier Inc. All rights reserved.)
Databáze:	MEDLINE
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