Margaret A. Weil

Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs

Katherine P. Ponder, John R. Melniczek, Lingfei Xu et al. · Proceedings of the National Academy of Sciences · 2002 · 169 citations · Full text

Enzyme Replacement Therapy in Feline Mucopolysaccharidosis I

Emil Kakkis, Edward H. Schuchman, Xingxuan He et al. · Molecular Genetics and Metabolism · 2001 · 89 citations

Enzyme Replacement Therapy, Pathogenesis, Veterinary Science +3

Transduction of Hepatocytes after Neonatal Delivery of a Moloney Murine Leukemia Virus Based Retroviral Vector Results in Long-Term Expression of β-Glucuronidase in Mucopolysaccharidosis VII Dogs

Lingfei Xu, Mark E. Haskins, John R. Melniczek et al. · Molecular Therapy · 2002 · 59 citations · Full text

Gene Therapy Ameliorates Cardiovascular Disease in Dogs With Mucopolysaccharidosis VII

Meg M. Sleeper, Benedetta Elena Fornasari, N. Matthew Ellinwood et al. · Circulation · 2004 · 47 citations · Full text

Gene Transfer of Low Levels of β-Glucuronidase Corrects Hepatic Lysosomal Storage in a Large Animal Model of Mucopolysaccharidosis VII

John H. Wolfe, Mark S. Sands, Noam Y. Harel et al. · Molecular Therapy · 2000 · 35 citations · Full text