Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease

Pompe disease is an autosomal recessive disorder caused by a deficiency of acid α-glucosidase (GAA; EC 3.2.1.20) and the resultant progressive lysosomal accumulation of glycogen in skeletal and cardiac muscles. Enzyme replacement therapy using recombinant human GAA (rhGAA) has proven beneficial in a...

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Main Authors: Clayton, Nicholas P, Nelson, Carol A, Weeden, Timothy, Taylor, Kristin M, Moreland, Rodney J, Scheule, Ronald K, Phillips, Lucy, Leger, Andrew J, Cheng, Seng H, Wentworth, Bruce M
Formato: Artigo
Idioma:Inglês
Publicado: Nature Publishing Group 2014
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Original Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC4217081/
https://ncbi.nlm.nih.gov/pubmed/25350581
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2014.57
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