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White matter disease: Targeted aspartoacylase gene therapy reverts Canavan disease – Nature.com


Nature.com

White matter disease: Targeted aspartoacylase gene therapy reverts Canavan disease
Nature.com
On the basis of these findings, the researchers designed a gene therapy approach to specifically deliver functional ASPA to oligodendrocytes. “Our approach employed somatic gene transfer using recombinant adeno-associated virus (AAV),” comments …

2017-11-24 NEWS
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