Tamar R. Grossman

This study shows that antisense oligonucleotides targeting VCP reduce disease pathology and improve muscle function in cell and mouse models of multisystem proteinopathy.

This study shows that ASO-mediated Gys1 knockdown in a mouse model of Pompe disease reduces glycogen accumulation and improves motor function, both alone and in combination with ERT.

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