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

Authors

Paola Leone, CNS Gene Therapy Center Department of Neurosurgery Thomas Jefferson University Philadelphia PA 19107 USAFollow
Christopher G Janson, Cooper University Hospital, Departments of Neurology and Molecular Genetics Camden, NJ USA
Scott J McPhee
Matthew J During, Ohio State University Comprehensive Cancer Center, Comprehensive Cancer Center Columbus, OH USA

Document Type

Article

Publication Date

8-1-1999

Comments

This article has been peer reviewed and is published in BMC Current Opinion in Molecular Therapeutics Volume 1, Issue 4, 1999, Pages 487-492. The published version is available at . Copyright © BioMed Central Ltd.

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.

Recommended Citation

Leone, Paola; Janson, Christopher G; McPhee, Scott J; and During, Matthew J, "Global CNS gene transfer for a childhood neurogenetic enzyme deficiency: Canavan disease." (1999). Department of Neurosurgery Faculty Papers. Paper 8.
https://jdc.jefferson.edu/neurosurgeryfp/8

PubMed ID

11713764