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
APA
Chicago
ICMJE
MLA
NLM
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 H, Fredrickson KB, Das S, Kishnani PS, Sun B. 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. 2013 Jul;109(3):312–4.
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.
Yi H, Fredrickson KB, Das S, Kishnani PS, Sun B. 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. 2013 Jul;109(3):312–314.
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