Gene Therapy
University of California, Department of Biological Chemistry describes research in gene therapy
Published in Gene Therapy Weekly, May 31st, 2007
New investigation results, "Lysosomal accumulation of SCMAS (subunit c of mitochondrial ATP synthase) in neurons of the mouse model of mucopolysaccharidosis III B," are detailed in a study published in Molecular Genetics and Metabolism. According to recent research from the United States, "The neurodegenerative disease MPS III B (Sanfilippo syndrome type B) is caused by mutations in the gene encoding the lysosomal enzyme alpha-N-acetylglucosaminidase, with a resulting block in heparan sulfate degradation. A mouse model with disruption of the Naglu gene allows detailed study of brain pathology."
"In contrast to somatic cells, which accumulate primarily heparan...
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Source: Gene Therapy Weekly (2007-05-31)
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