NeuroscienceNeurotherapeuticsPeer-reviewed
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…·Berge Arakel Minassian
This study uses AAV-delivered CRISPR/Cas9 to target Gys1 in mouse models of neurodegenerative diseases, reducing polyglucosan bodies and neuroinflammation.
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