Tay-Sachs Disease

- Cell Therapy Approaches

Tay-Sachs disease is a neurodegenerative metabolic disease caused by a mutation in the HEXA gene, which encodes the alpha-subunit of the lysosomal enzyme beta-N-acetylhexosaminidase. As a result, the enzyme exhibits impaired function, leading to ganglioside accumulation in neurons and eventually to progressive neuronal death in the brain and spinal cord. To date, there is no treatment or cure for Tay-Sachs disease.

Cell Therapy Approaches

In patients with enzymatic abnormalities and metabolic storage disorders, cell therapies enable synthesis of normal functional enzymes that may be capable of circulating and improving cellular functions in the native tissues.

Neuronal stem cells (NSCs) delivered into the central nervous system, via the spinal canal, may replace and/or repair the damaged neuronal tissues.

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Tay-Sachs Disease

- Cell Therapy Approaches

Cell Therapy Approaches

Cell Therapies for the treatment of

Modeling of

Affected or Therapy-targeted Cells/Compartments for

Literature related to

Welcome to LifeMap Discovery

Watch the introductory video to learn more about our database of embryonic development for stem cell research and regenerative medicine

Tay-Sachs Disease - Cell Therapy Approaches

Cell Therapy Approaches

Cell Therapies for the treatment of

Modeling of

Affected or Therapy-targeted Cells/Compartments for

Literature related to

Welcome to
LifeMap Discovery

Tay-Sachs Disease

- Cell Therapy Approaches