Central Nervous System Diseases
Studies from Mackay Memorial Hospital Have Provided New Data on Central Nervous System Diseases
Published in Gene Therapy Weekly, March 29th, 2012
2012 MAR 29 -- A report, "CNS-targeted AAV5 gene transfer results in global dispersal of vector and prevention of morphological and function deterioration in CNS of globoid cell leukodystrophy mouse model," is newly published data in Molecular Genetics and Metabolism. "Globoid cell leukodystrophy (GLD) is a devastating lysosomal storage disease caused by deficiency of the enzyme galactocerebrosidase (GALC). Currently, there is no definite cure for GLD," scientists writing in the journal Molecular Genetics and Metabolism report.
"Several attempts with CNS-directed gene therapy in twitcher mice (a murine model of GLD) demonstrated restricted...
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Source: Gene Therapy Weekly (2012-03-29)
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