Metabolic Disease
Adeno-associated virus type 1 corrects storage lesions in enzyme-deficient mice
Published in Gene Therapy Weekly, July 24th, 2003
According to published research from the United States, "Inherited metabolic disorders that affect the central nervous system typically result in pathology throughout the brain; thus, gene therapy strategies need to achieve widespread delivery. We previously found that although intraventricular injection of the neonatal mouse brain with adeno-associated virus serotype 2 (AAV2) results in dispersed gene delivery, many brain structures were poorly transduced. This limitation may be overcome by using different AAV serotypes because the capsid proteins use different cellular receptors for...
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Source: Gene Therapy Weekly (2003-07-24)
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