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PRECLINICAL MODELS  |  RARE DISEASES  |  LYSOSOMAL DISEASES

Lysosomal diseases / Gaucher's disease

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.