EMBRYONIC DEVELOPMENT & STEM CELL COMPENDIUM
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All Diseases > Disease Card

Sandhoff Disease, Infantile, Juvenile, and Adult Forms
 - 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.

Sandhoff Disease, Infantile, Juvenile, and Adult Forms