| Global CNS gene transfer for a childhood neurogenetic enzyme deficiency: Canavan disease. | |
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MedLine Citation:
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PMID: 11713764 Owner: NLM Status: MEDLINE |
Abstract/OtherAbstract:
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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. |
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Authors:
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P Leone; C G Janson; S J McPhee; M J During |
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Publication Detail:
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Type: Journal Article; Review |
Journal Detail:
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Title: Current opinion in molecular therapeutics Volume: 1 ISSN: 1464-8431 ISO Abbreviation: Curr. Opin. Mol. Ther. Publication Date: 1999 Aug |
Date Detail:
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Created Date: 2001-11-20 Completed Date: 2002-01-02 Revised Date: 2008-11-21 |
Medline Journal Info:
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Nlm Unique ID: 100891485 Medline TA: Curr Opin Mol Ther Country: England |
Other Details:
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Languages: eng Pagination: 487-92 Citation Subset: IM |
Affiliation:
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CNS Gene Therapy Center, Department of Neurosurgery, Thomas Jefferson University, Philadelphia, PA 19107, USA. Paola.Leone@mail.tju.edu |
Export Citation:
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APA/MLA Format Download EndNote Download BibTex |
| MeSH Terms | |
Descriptor/Qualifier:
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Acetates
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therapeutic use Acetazolamide / therapeutic use Amidohydrolases / deficiency, genetics*, physiology Animals 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 Child, Preschool Cholesterol / administration & dosage, analogs & derivatives* Combined Modality Therapy Cytomegalovirus / genetics DNA, Recombinant / administration & dosage, therapeutic use Dependovirus / genetics Female Fetal Diseases / diagnosis, genetics Gene Therapy* Genetic Vectors / administration & dosage, genetics, therapeutic use Glycerophospholipids / administration & dosage Humans Infant Injections, Intraventricular Liposomes / administration & dosage Male Mitochondria / metabolism, pathology Nerve Tissue Proteins / deficiency, genetics*, physiology Osmotic Pressure Phosphatidylethanolamines* Prenatal Diagnosis Primates Promoter Regions, Genetic Protamines / administration & dosage Rats |
| Chemical | |
Reg. No./Substance:
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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 3.5.1.15/aspartoacylase |
From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine
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