Jason A Metcalf
Affiliation: Washington University School of Medicine
- Mechanism of shortened bones in mucopolysaccharidosis VIIJason A Metcalf
Department of Medicine, Washington University School of Medicine, Campus Box 8125, 660 South Euclid Avenue, Saint Louis, MO 63110, USA
Mol Genet Metab 97:202-11. 2009..This suggests that accumulation of C4S in the growth plate leads to reduced expression of LIF and reduced STAT3 tyrosine phosphorylation, which results in reduced chondrocyte proliferation and ultimately shortened bones...
- A self-inactivating gamma-retroviral vector reduces manifestations of mucopolysaccharidosis I in miceJason A Metcalf
Department of Medicine, Washington University School of Medicine, St Louis, Missouri, USA
Mol Ther 18:334-42. 2010..A SIN gamma-RV vector ameliorates clinical manifestations of MPS I in mice and should be safer than an LTR-intact gamma-RV...
- Neonatal gene therapy with a gamma retroviral vector in mucopolysaccharidosis VI catsKatherine P Ponder
Department of Internal Medicine, Washington University School of Medicine, St Louis, Missouri 63110, USA
Mol Ther 20:898-907. 2012..This demonstrates that therapeutic expression of a functional sulfatase protein can be achieved with neonatal gene therapy using a gamma RV, but some aspects of bone disease remain difficult to treat...
- Upregulation of elastase activity in aorta in mucopolysaccharidosis I and VII dogs may be due to increased cytokine expressionJason A Metcalf
Department of Internal Medicine, Washington University School of Medicine, St Louis, MO 63110, USA
Mol Genet Metab 99:396-407. 2010..Inhibitors of these enzymes or these cytokine-induced signal transduction pathways might reduce aortic disease in patients with MPS...