Exp. antisense treatment for Pompe disease

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jonmoulton
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Exp. antisense treatment for Pompe disease

Post by jonmoulton » Wed Oct 29, 2014 3:58 pm

Here's a nice preclinical report of antisense as a potential therapeutic.

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

http://www.nature.com/mtna/journal/v3/n10/full/mtna201457a.html

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