Targeting Gys1 with AAV-SaCas9 decreases pathogenic polyglucosan bodies and neuroinflammation in Adult Polyglucosan Body and Lafora disease mouse models
Emrah Gumusgoz, Dikran Richard Guisso, Sahba Kasiri, Jun Wu, Matthew Dear, Brandy Verhalen, Silvia Nitschke, Sharmistha Mitra, Felix Nitschké, Berge Arakel Minassian
This study uses AAV-delivered CRISPR/Cas9 to target Gys1 in mouse models of neurodegenerative diseases, reducing polyglucosan bodies and neuroinflammation.
Neurotherapeutics · Apr 1, 2021Peer-reviewedNeuroscience