Intracellular Delivery of β-Galactosidase Enzyme Using Arginase-Responsive Dextran Sulfate/Poly-l-arginine Capsule for Lysosomal Storage Disorder.

ABSTRACT: β-Galactosidase (β-gal) is one of the important lysosomal enzymes that is involved in the breakdown of glycosphingolipids (e.g., GM1 ganglioside), and its deficiency leads to GM1 Gangliosidosis, a lysosomal storage disorder (LSD). Intracellular delivery of β-gal is one of the preferable methods to treat this kind of LSDs. However, it cannot permeate the cell membrane due to its intricate macromolecular nature, low stability, and degradation by endogenous proteases. To this end, we report efficient intracellular delivery of β-gal via arginase-responsive dextran sulfate/poly-l-arginine polymer capsules (DS/PA capsules). The therapeutic activity of β-gal enzyme has been assessed in two gene-deficient diseased cell lines, SV (β-galactosidase gene-deficient mouse fibroblast) and R201C (deficien

SUBMITTER: Gupta M 

PROVIDER: S-EPMC6044979 | biostudies-literature | 2017 Dec

REPOSITORIES: biostudies-literature