IN VITRO models​

​

Primary culture of central neurons injured with specific toxins:

• Gaucher’s disease: primary mesencephalic neurons injured with a GBA inhibitor (CBE)

• GM1 gangliosidosis: primary cortical neurons injured with a GLB1 inhibitor (hirsutine)

• Krabbe disease: primary cortical neurons and oligodendrocytes injured with Psychosine

• Hurler's syndrome: primary cortical neurons injured with an IDUA inhibitor

​

Culture of SH-SY5Y cell lines harboring point mutations:

• Gaucher’s disease: L444P mutation on GBA

​

Other models currently under development.

​

​