Felix Nitschké

This study shows that Gys1 antisense therapy reduces Lafora body formation and neuroinflammation in mouse models of Lafora disease.

This study uses AAV-delivered CRISPR/Cas9 to target Gys1 in mouse models of neurodegenerative diseases, reducing polyglucosan bodies and neuroinflammation.

This study shows that RBCK1 deficiency in mice leads to brain polyglucosan accumulation and neurodegeneration, similar to Lafora disease but with distinct regional and subcellular features.