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Significance Lysosomal storage disorders (LSDs) are one of the most prevalent inherited pediatric conditions. Although some LSDs were described over a century ago, we understand little about how specific cells contribute to pathogenesis. Most LSDs are caused by a lysosomal enzyme deficiency. Lysosomal enzymes can be secreted from one cell and correct neighboring cells (cross-correction). Cross-correction prevents cell-limited expression or deletion of lysosomal enzymes, making it impossible to determine the role of specific cells in LSDs. Here we describe a method to eliminate cross-correction while maintaining enzymatic function. We used this method to determine the role of specific cells (myelin-producing) in Krabbe disease. This same method could be used to determine the role of specific cells in most other LSDs.

Cell-autonomous expression of the acid hydrolase galactocerebrosidase