Tay-Sachs disease brain cells in culture: mobilization of stored GM2 after concanavalin A-mediated uptake of hexosaminidase A.

A human Tay-Sachs disease (TSD) fetal-brain-cell line is a useful model for the disease since the cells lack hexosaminidase A and accumulate the ganglioside, GM2. This brain-cell line was used to assess the effect of hexosaminidase A treatment on GM2 storage material. Entry of placental hexosaminidase A into the cells was obtained by pretreatment of the cultures with concanavalin A. Cells were analyzed periodically during six days. During the course of the experiment, GM2 in the cells decreased by approximately 50%. A substantial amount of hexosaminidase A was maintained in the cultures throughuot the experiment. This strategy was successful in mobilizing stored GM2 in TSD brain-cell cultures. Therefore, the activating factor needed for hexosaminidas A activity must be present in TSD-cultured brain cells.