Protocol for chemogenetic activation of basal ganglia D1-MSNs and behavioral assessments in a primate Parkinson’s disease model
Brief intro:
- Author: Yefei Chen, Zexuan Hong, Ting Yan, Yuyuan Zhu, Jianbang Lin, Taian Liu, Ming Guo, Mengqi Li, Shijing Feng, Ronghui Li, Jianqing Zhang, Siyang Chen, Yi Yang, Yuantao Li, Zhonghua Lu,
- Journal: STAR Protocols
- Publication Date: 2024 Dec 20
Abstract
A circuit-based gene therapy strategy for Parkinson’s disease (PD) has been shown to significantly reverse core symptoms in both murine and primate PD models. Here, we present a comprehensive workflow to specifically manipulate dopamine receptor D1-expressing medium spiny neurons by retrograde adeno-associated virus (AAV) transduction and chemogenetic activation using a designer toolkit. We describe steps for AAV injections and PD primate model induction. We then detail behavioral measurements to assess the therapeutic efficacy of the therapy for motor symptoms.
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PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
