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

Publication ,  Journal Article
Yi, H; Fredrickson, KB; Das, S; Kishnani, PS; Sun, B
Published in: Mol Genet Metab
July 2013

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.

Duke Scholars

Published In

Mol Genet Metab

DOI

EISSN

1096-7206

Publication Date

July 2013

Volume

109

Issue

3

Start / End Page

312 / 314

Location

United States

Related Subject Headings

  • RNA Interference
  • Muscle, Skeletal
  • Muscle Proteins
  • Mice, Knockout
  • Mice
  • Membrane Proteins
  • Lysosomes
  • Humans
  • Glycogen Storage Disease Type II
  • Glycogen
 

Citation

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Yi, H., Fredrickson, K. B., Das, S., Kishnani, P. S., & Sun, B. (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), 312–314. https://doi.org/10.1016/j.ymgme.2013.05.004
Yi, Haiqing, Keri B. Fredrickson, Stuti Das, Priya S. Kishnani, and Baodong Sun. “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, no. 3 (July 2013): 312–14. https://doi.org/10.1016/j.ymgme.2013.05.004.
Yi, Haiqing, et al. “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, vol. 109, no. 3, July 2013, pp. 312–14. Pubmed, doi:10.1016/j.ymgme.2013.05.004.
Journal cover image

Published In

Mol Genet Metab

DOI

EISSN

1096-7206

Publication Date

July 2013

Volume

109

Issue

3

Start / End Page

312 / 314

Location

United States

Related Subject Headings

  • RNA Interference
  • Muscle, Skeletal
  • Muscle Proteins
  • Mice, Knockout
  • Mice
  • Membrane Proteins
  • Lysosomes
  • Humans
  • Glycogen Storage Disease Type II
  • Glycogen