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

• Main Authors: Nicholas P Clayton (Author), Carol A Nelson (Author), Timothy Weeden (Author), Kristin M Taylor (Author), Rodney J Moreland (Author), Ronald K Scheule (Author), Lucy Phillips (Author), Andrew J Leger (Author), Seng H Cheng (Author), Bruce M Wentworth (Author)
• Formato: Livro
• Publicado em: Elsevier, 2014-01-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: Connect to this object online.

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...