Protocol for chemogenetic activation of basal ganglia D1-MSNs and behavioral assessments in a primate Parkinson’s disease model
简介:
- 作者: 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,
- 杂志: STAR Protocols
- 出版日期: 2024 Dec 20
摘要
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.
关于派真
作为一家专注于AAV 技术十余年，深耕基因治疗领域的CRO&CDMO，派真生物可提供从载体设计、构建到 AAV、慢病毒和 mRNA 服务的一站式解决方案。凭借深厚的技术实力、卓越的运营管理和高标准的服务交付，我们为全球客户提供一站式CMC解决方案，包括从早期概念验证、成药性评估到IIT、IND及BLA的各个阶段。
凭借我们独立知识产权的π-alphaTM 293 细胞AAV高产技术平台，我们能将AAV产量提高多至10倍，每批次产量可达1×10¹⁷vg，以满足多样化的商业化和临床项目需求。此外，我们定制化的mRNA和脂质纳米颗粒（LNP）产品及服务覆盖药物和疫苗开发的各个阶段，从研发到符合GMP的生产，提供端到端的一站式解决方案。
