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…·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.