Gene Therapy
Data from University of Padova Provide New Insights into Gene Therapy
Published in Gene Therapy Weekly, May 10th, 2012
2012 MAY 10 -- Investigators discuss in "Encapsulated engineered myoblasts can cure Hurler syndrome: preclinical experiments in the mouse model" new findings in Gene Therapy. "Mucopolysaccharidosis type I (MPSI) is an autosomic recessive, lysosomal storage disorder due to the deficit of the enzyme a-L-iduronidase (IDUA). The disease accounts for a general impairment of tissue and organ functions, mainly including heart disease, corneal clouding, organomegaly, skeletal malformations and joint stiffness," scientists in Padova, Italy report.
"Neurological deterioration affects the severe forms. Both haemopoietic stem cell transplantation and enzyme replacement...
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Source: Gene Therapy Weekly (2012-05-10)
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