Gene therapy for lysosomal storage disease: a no-brainer? Transplants of fibroblasts secreting high levels of beta-glucuronidase decrease lesions in the brains of mice with Sly syndrome, a lysosomal storage disease.
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D000818:Animals; D001921:Brain / Q000378:metabolism; D005347:Fibroblasts / Q000378:metabolism / Q000637:transplantation; D015316:Genetic Therapy; D005966:Glucuronidase / Q000235:genetics* / Q000378:metabolism; D006025:Glycosaminoglycans / Q000378:metabolism; D006801:Humans; D051379:Mice; D016538:Mucopolysaccharidosis VII / Q000628:therapy*; D017527:Receptor, IGF Type 2 / Q000378:metabolism
D006025:Glycosaminoglycans; D017527:Receptor, IGF Type 2; D005966:Glucuronidase