Editorial Note: The Pharmacological Chaperone AT2220 Increases Recombinant Human Acid α-Glucosidase Uptake and Glycogen Reduction in a Mouse Model of Pompe Disease

Abstract
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Taxonomy
TopicsLysosomal Storage Disorders Research · Glycogen Storage Diseases and Myoclonus · Carbohydrate Chemistry and Synthesis
After publication of this article [1], concerns were raised about the interpretation of Fig1B. Specifically, the fourth sentence of the Results sub-section titled AT2220 Stabilized rhGAA, Preventing Denaturation and Loss of Activity, reports a loss of GAA activity with a half-life of 3–4 hours in Fig 1B. However, upon editorial assessment the PLOS One Editors consider the data in Fig 1B to appear to be consistent with a GAA half-life of 13–14 hours.
Additionally, it is noted that the primary data underlying results in this article [1] are not included with the published article.
The authors did not respond to the journal’s communications.
The PLOS One Editors issue this Editorial Note to inform readers of the above discrepancy, which does not appear to affect the article’s main results and conclusions.
The reference list from the paper itself. Each links out to its DOI / PubMed record.
- 1Khanna R, Flanagan JJ, Feng J, Soska R, Frascella M, Pellegrino LJ, et al. The pharmacological chaperone AT 2220 increases recombinant human acid α-glucosidase uptake and glycogen reduction in a mouse model of Pompe disease. P Lo S One. 2012;7(7):e 40776. doi: 10.1371/journal.pone.0040776 22815812 PMC 3399870 · doi ↗ · pubmed ↗
