Gene Therapy
Reports outline gene therapy research from University of Paris
Published in Pharma Law Weekly, February 3rd, 2009
"Glycogen storage disease type II (GSDII) or Pompe disease is an autosomal recessive disorder caused by defects in the acid alpha-glucosidase gene, which leads to lysosomal glycogen accumulation and enlargement of the lysosomes mainly in cardiac and muscle tissues, resulting in fatal hypertrophic cardiomyopathy and respiratory failure in the most severely affected patients. Enzyme replacement therapy has already proven to be beneficial in this disease, but correction of pathology in skeletal muscle still remains a challenge," scientists writing in the journal Human Molecular Genetics report.
"As substrate deprivation was successfully used to improve the phenotype...
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Source: Pharma Law Weekly (2009-02-03)
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