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Article

Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease

1 Jul 2000

Abstract excerpt

With the ultimate goal of developing safe and effective in vivo gene therapy for the treatment of Canavan disease and other neurological disorders, we developed a non-viral lipid-entrapped, polycation-condensed delivery system (LPD) for central nervous system gene transfer, in conjunction with adeno-associated virus (AAV)-based plasmids containing recombinant aspartoacylase (ASPA). The gene delivery system was...

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PMID: 10894213
DOI: 10.1002/1531-8249(200007)48:1<27::aid-ana6>3.0.co;2-6

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Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan diseasePMID 10894213Read ↗

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