Pompe Disease: Block making Glycogen Synthase 1 for Substrate Reduction

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Jon Moulton
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Pompe Disease: Block making Glycogen Synthase 1 for Substrate Reduction

Clayton NP, Nelson CA, Weeden T, Taylor KM, Moreland RJ, Scheule RK, Phillips L, Leger AJ, Cheng SH, Wentworth BM. Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease. Mol Ther Nucleic Acids. 2014 Oct 28;3:e206. doi: 10.1038/mtna.2014.57.