| First Author | Yi H | Year | 2013 |
| Journal | Mol Genet Metab | Volume | 109 |
| Issue | 3 | Pages | 312-4 |
| PubMed ID | 23726947 | Mgi Jnum | J:203218 |
| Mgi Id | MGI:5525200 | Doi | 10.1016/j.ymgme.2013.05.004 |
| Citation | Yi H, et al. (2013) Stbd1 is highly elevated in skeletal muscle of Pompe disease mice but suppression of its expression does not affect lysosomal glycogen accumulation. Mol Genet Metab 109(3):312-4 |
| abstractText | 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. |
