Faculty Research 1990 - 1999
Gene therapy for murine mucopolysaccharidosis type VII.
Document Type
Article
Publication Date
1997
Keywords
Animal, Bone-Marrow-Transplantation, Disease-Models-Animal, Gene-Therapy, Genetic-Vectors, Glucuronidase: se, Hematopoiesis, Mice, Mucopolysaccharidosis-VII: pa, th, ve, Rodent-Diseases: th
First Page
352
Last Page
360
JAX Source
Neuromuscul Disord 1997 Jul;7(5):352-60
Abstract
Mucopolysaccharidosis type VII (MPS VII) is caused by a deficiency in the lysosomal enzyme beta-glucuronidase resulting in the accumulation of undegraded glycosaminoglycans in many tissues. A murine model of MPS VII shares many of the clinical, biochemical and histopathological features of human MPS VII and has provided an opportunity to study novel therapeutic approaches in a system with a uniform genetic background. Retroviral mediated gene therapy directed to the hematopoietic system or to artificial neo-organs resulted in low levels of enzyme in several tissues and reduced lysosomal storage in the liver and spleen. Partial correction of the disease in the eye was observed following an intravitreal injection of recombinant adenovirus. Neither retroviral nor adenoviral mediated gene transfer techniques resulted in a systemic reduction of lysosomal storage. Here we discuss several novel gene transfer approaches designed to increase the systemic levels of beta-glucuronidase in the MPS VII mouse.
Recommended Citation
Sands MS,
Wolfe JH,
Birkenmeier EH,
Barker JE,
Vogler C,
Sly WS,
Okuyama T,
Freeman B,
Nicholes A,
Muzyczka N,
Chang PL,
Axelrod HR.
Gene therapy for murine mucopolysaccharidosis type VII. Neuromuscul Disord 1997 Jul;7(5):352-60