Document Detail

Global CNS gene transfer for a childhood neurogenetic enzyme deficiency: Canavan disease.
MedLine Citation:
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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Publication Detail:
Type:  Journal Article; Review    
Journal Detail:
Title:  Current opinion in molecular therapeutics     Volume:  1     ISSN:  1464-8431     ISO Abbreviation:  Curr. Opin. Mol. Ther.     Publication Date:  1999 Aug 
Date Detail:
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    
Other Details:
Languages:  eng     Pagination:  487-92     Citation Subset:  IM    
CNS Gene Therapy Center, Department of Neurosurgery, Thomas Jefferson University, Philadelphia, PA 19107, USA.
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MeSH Terms
Acetates / therapeutic use
Acetazolamide / therapeutic use
Amidohydrolases / deficiency,  genetics*,  physiology
Aspartic Acid / analogs & derivatives*,  metabolism*,  physiology
Brain / enzymology*,  pathology
Brain Edema / etiology,  physiopathology
Calcium Compounds
Canavan Disease / diagnosis,  embryology,  enzymology,  genetics,  pathology,  physiopathology,  therapy*
Child, Preschool
Cholesterol / administration & dosage,  analogs & derivatives*
Combined Modality Therapy
Cytomegalovirus / genetics
DNA, Recombinant / administration & dosage,  therapeutic use
Dependovirus / genetics
Fetal Diseases / diagnosis,  genetics
Gene Therapy*
Genetic Vectors / administration & dosage,  genetics,  therapeutic use
Glycerophospholipids / administration & dosage
Injections, Intraventricular
Liposomes / administration & dosage
Mitochondria / metabolism,  pathology
Nerve Tissue Proteins / deficiency,  genetics*,  physiology
Osmotic Pressure
Prenatal Diagnosis
Promoter Regions, Genetic
Protamines / administration & dosage
Reg. No./Substance:
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

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine

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