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

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

STAR Protocols (Dec 2024)

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

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

Affiliations

Kan Yang: 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; Corresponding author
Tianshu Li: 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
Yixiao Geng: 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
Ru Zhang: College of Materials and Chemical Engineering, Hunan Institute of Engineering, Xiangtan, Hunan 411104 , China
Zhankui Xu: College of Materials and Chemical Engineering, Hunan Institute of Engineering, Xiangtan, Hunan 411104 , China
Jun Wu: 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
Yiting Yuan: 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
Yuefang Zhang: 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
Zilong Qiu: 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; Corresponding author
Fei Li: 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; Corresponding author

Journal volume & issue: Vol. 5, no. 4
p. 103344

Abstract

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Summary: 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 : Publisher’s note: Undertaking any experimental protocol requires adherence to local institutional guidelines for laboratory safety and ethics.

Published in STAR Protocols

ISSN: 2666-1667 (Online)
Publisher: Elsevier
Country of publisher: United States
LCC subjects: Science: Science (General)
Website: https://star-protocols.cell.com/

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