Global CNS gene transfer for a childhood neurogenetic enzyme deficiency: Canavan disease

Paola Leone, Christopher G Janson, Scott J. McPhee, Matthew J. During

Research output: Contribution to journalReview articlepeer-review

39 Scopus citations

Abstract

The neurogenetic prototypic disease on which we chose to test our gene therapy strategy is Canavan disease (CD). CD is an autosomal recessive leukodystrophy associated with spongiform degeneration of the brain. At present the disease is uniformly fatal in affected probands. CD is characterized by mutations in the aspartoacylase (ASPA) gene, resulting in loss of enzyme activity. In this review, recent evidence is summarized on the etiology and possible treatments for CD. In particular, we discuss two gene delivery systems representing recent advances in both viral and liposome technology: a novel cationic liposome-polymer-DNA (LPD) complex, DCChol/DOPE- protamine, as well as recombinant adeno-associated virus (AAV) vectors.

Original languageEnglish (US)
Pages (from-to)487-492
Number of pages6
JournalCurrent Opinion in Molecular Therapeutics
Volume1
Issue number4
StatePublished - Sep 15 1999

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