Antibody-mediated enzyme replacement therapy targeting both lysosomal and cytoplasmic glycogen in Pompe disease

@article{Yi2017AntibodymediatedER,
  title={Antibody-mediated enzyme replacement therapy targeting both lysosomal and cytoplasmic glycogen in Pompe disease},
  author={Haiqing Yi and Tao Sun and Dustin Armstrong and Scott Borneman and Chunyu Yang and Stephanie Austin and Priya Kishnani and Baodong Sun},
  journal={Journal of Molecular Medicine},
  year={2017},
  volume={95},
  pages={513-521}
}
Pompe disease is characterized by accumulation of both lysosomal and cytoplasmic glycogen primarily in skeletal and cardiac muscles. Mannose-6-phosphate receptor-mediated enzyme replacement therapy (ERT) with recombinant human acid α-glucosidase (rhGAA) targets the enzyme to lysosomes and thus is unable to digest cytoplasmic glycogen. Studies have shown that anti-DNA antibody 3E10 penetrates living cells and delivers “cargo” proteins to the cytosol or nucleus via equilibrative nucleoside… CONTINUE READING