|Global CNS gene transfer for a childhood neurogenetic enzyme deficiency: Canavan disease.|
|PMID: 11713764 Owner: NLM Status: MEDLINE|
|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.|
|P Leone; C G Janson; S J McPhee; M J During|
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|Type: Journal Article; Review|
|Title: Current opinion in molecular therapeutics Volume: 1 ISSN: 1464-8431 ISO Abbreviation: Curr. Opin. Mol. Ther. Publication Date: 1999 Aug|
|Created Date: 2001-11-20 Completed Date: 2002-01-02 Revised Date: 2008-11-21|
Medline Journal Info:
|Nlm Unique ID: 100891485 Medline TA: Curr Opin Mol Ther Country: England|
|Languages: eng Pagination: 487-92 Citation Subset: IM|
|CNS Gene Therapy Center, Department of Neurosurgery, Thomas Jefferson University, Philadelphia, PA 19107, USA. Paola.Leone@mail.tju.edu|
|APA/MLA Format Download EndNote Download BibTex|
Acetazolamide / therapeutic use
Amidohydrolases / deficiency, genetics*, physiology
Aspartic Acid / analogs & derivatives*, metabolism*, physiology
Brain / enzymology*, pathology
Brain Edema / etiology, physiopathology
Canavan Disease / diagnosis, embryology, enzymology, genetics, pathology, physiopathology, therapy*
Cholesterol / administration & dosage, analogs & derivatives*
Combined Modality Therapy
Cytomegalovirus / genetics
DNA, Recombinant / administration & dosage, therapeutic use
Dependovirus / genetics
Fetal Diseases / diagnosis, genetics
Genetic Vectors / administration & dosage, genetics, therapeutic use
Glycerophospholipids / administration & dosage
Liposomes / administration & dosage
Mitochondria / metabolism, pathology
Nerve Tissue Proteins / deficiency, genetics*, physiology
Promoter Regions, Genetic
Protamines / administration & dosage
|0/1,2-dioleoyl-glycero-3-phosphatidyl ethanolamine; 0/Acetates; 0/Calcium Compounds; 0/DNA, Recombinant; 0/Glycerophospholipids; 0/Liposomes; 0/Nerve Tissue Proteins; 0/Phosphatidylethanolamines; 0/Protamines; 137056-72-5/3-(N-(N',N'-dimethylaminoethane)carbamoyl)cholesterol; 56-84-8/Aspartic Acid; 57-88-5/Cholesterol; 59-66-5/Acetazolamide; 62-54-4/calcium acetate; 997-55-7/N-acetylaspartate; EC 3.5.-/Amidohydrolases; EC 18.104.22.168/aspartoacylase|
From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine
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