Research
Enhancer-based AAV approach for selective AADC delivery reduces motor symptoms and dyskinesia in Parkinson’s mouse models
Cell Reports · By Ananya Chowdhury, Alex Fraser, Meagan A. Quinlan, Max Departee, Naz Taskin, John K. Mich, Victoria Omstead, Nathaly Lerma, Ximena Opitz-Araya, Alex C. Hughes, Emily Kussick, Refugio Martinez, Melissa Reding, Elizabeth Liang, Lyudmila Shulga, Dean Rette, Cindy Huang, Brittny Casian, Madison Leibly, Olivia Helback, Tyler Barcelli, Toren Wood, Nicholas Uribe, Colin Bacon, Jessica Bowlus, Dakota Newman, Rana Kutsal, Shannon Khem, Nicholas Donadio, Shenqin Yao, Kara Ronellenfitch, Vonn Wright, Kathryn Gudsnuk, Greg D. Horwitz, Boaz P. Levi, Ed S. Lein, Jonathan T. Ting & Tanya L. Daigle ·
The Brief
- Chowdhury et al. developed enhancer-driven AAV vectors that achieve cell type-specific AADC expression in mice and non-human primates.
- In Parkinson's disease mouse models, targeting different cell populations produced differing behavioral effects.
- The study reports that the approach reduces motor symptoms and dyskinesia in these models.
Why it matters
It shows that cell-type-specific gene delivery to the brain can be achieved with enhancer-based AAVs, which could allow more precise AADC gene therapy for Parkinson's and help explain why targeting different cell populations yields different outcomes.
Who’s affected
- researchers in gene therapy and viral vector design
- neuroscientists studying Parkinson's disease and motor symptoms
- #gene therapy
- #parkinson's disease
- #aav
- #aadc
- #enhancers
- #dyskinesia
Chowdhury et al. develop enhancer-driven AAVs that achieve cell type-specific AADC expression in both mice and non-human primates. Targeting distinct cell populations produces differential behavioral effects across Parkinson’s disease mouse models, demonstrating the importance of…