Robust nuclease-dead S. aureus dCas9-mediated alpha-synuclein knockdown in substantia nigra in a humanized mouse model of Parkinson's disease
C. Alejandra Morato Torres, Faria Zafar, Danuta Sastre, De’Angelo Hermesky, Max Yang Chen, Jocelyn Palafox Vazquez, Lei S. Qi, Deniz Kirik, Birgitt Schüle
This paper demonstrates that sadCas9-mediated CRISPRi reduces alpha-synuclein expression in a humanized mouse model of Parkinson's disease, showing long-term downregulation and attenuated immune response.
bioRxiv · Sep 5, 2023 · PreprintNeuroscience