Martin, M. del Pilar

disease protein expression mouse iysosomal brain enzyme storage level model tissue death gm 1 mice activity month correction murine gs mice organ result cell death caspase stress patient accumulation therapy response galactosialidosi macrophage phenotype ofthe purkinje neuron system disorder apoptosi transplantation marrow ppca expression vector blood activation function pathway gangliosidosi deficiency animal feature pathology ganglioside mouse model bone marrow transplantation wild-type antibody liver number substrate figure study suzuki purkinje cells analysis symptom motor involvement sample sandhoff gm 1 gangliosidosis lysosomal martin amelioration mechanism onset iysosomal storage promoter reticulum kidney control transfer precursor