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Mol Genet Metab. 2013 Jul;109(3):312-4. doi: 10.1016/j.ymgme.2013.05.004. Epub 2013 May 18.

Stbd1 is highly elevated in skeletal muscle of Pompe disease mice but suppression of its expression does not affect lysosomal glycogen accumulation.

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  • 1Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.


Previous studies strongly suggest that starch binding domain containing protein 1 (Stbd1) plays an important role in intracellular glycogen trafficking into lysosomes. We report here that Stbd1 expression is markedly increased in skeletal muscles but not in heart and liver of GAA-KO mice. An AAV2/9 vector expressing a Stbd1-specific shRNA effectively suppressed Stbd1 expression but did not alter lysosomal glycogen accumulation in the affected tissues of GAA-KO mice. Our results indicate that inhibition of Stbd1 does not appear to be an effective therapeutic approach for Pompe disease.

Copyright © 2013 Elsevier Inc. All rights reserved.

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