Sandhoff Disease

- Cell Therapy Approaches

Sandhoff disease is an inherited lysosomal lipid storage disorder caused by a deficiency of functional beta-hexosaminidases A and B, leading to ganglioside accumulation and resulting in the progressive deterioration of the central nervous system. Currently, Sandhoff disease is incurable.

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.

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Sandhoff 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

Sandhoff 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

Sandhoff Disease

- Cell Therapy Approaches