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Recombinant AAV-mediated gene transfer to the retina: gene therapy perspectives

Retinal degenerative diseases such as retinal macular degeneration and retinitis pigmentosa constitute a broad group of diseases that all share one critical feature, the progressive apoptotic loss of cells in the retina. There is currently no effective treatment available by which the course of thes... Full description

Journal Title: Gene Therapy Oct 2004, Vol.11(S1), pp.S26-32
Main Author: Rolling, F
Format: Electronic Article Electronic Article
Language: English
Subjects:
ID: ISSN: 09697128 ; E-ISSN: 14765462 ; DOI: 10.1038/sj.gt.3302366
Link: http://search.proquest.com/docview/218676687/?pq-origsite=primo
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title: Recombinant AAV-mediated gene transfer to the retina: gene therapy perspectives
format: Article
creator:
  • Rolling, F
subjects:
  • Animals–Genetics
  • Dependovirus–Methods
  • Genetic Therapy–Administration & Dosage
  • Genetic Vectors–Administration & Dosage
  • Humans–Therapy
  • Models, Animal–Methods
  • Recombinant Proteins–Methods
  • Retinal Diseases–Methods
  • Transduction, Genetic–Methods
  • Recombinant Proteins
ispartof: Gene Therapy, Oct 2004, Vol.11(S1), pp.S26-32
description: Retinal degenerative diseases such as retinal macular degeneration and retinitis pigmentosa constitute a broad group of diseases that all share one critical feature, the progressive apoptotic loss of cells in the retina. There is currently no effective treatment available by which the course of these disorders can be modified, and visual dysfunction often progresses to total blindness. Gene therapy represents an attractive approach to treating retinal degeneration because the eye is easily accessible and allows local application of therapeutic vectors with reduced risk of systemic effects. Furthermore, transgene expression within the retina and effects of treatments may be monitored by a variety of noninvasive examinations. An increasing number of strategies for molecular treatment of retinal disease rely on recombinant adeno-associated virus (rAAV) as a therapeutic gene delivery vector. Before rAAV-mediated gene therapy for retinal degeneration becomes a reality, there are a number of...
language: eng
source:
identifier: ISSN: 09697128 ; E-ISSN: 14765462 ; DOI: 10.1038/sj.gt.3302366
fulltext: fulltext
issn:
  • 09697128
  • 0969-7128
  • 14765462
  • 1476-5462
url: Link


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subjectAnimals–Genetics ; Dependovirus–Methods ; Genetic Therapy–Administration & Dosage ; Genetic Vectors–Administration & Dosage ; Humans–Therapy ; Models, Animal–Methods ; Recombinant Proteins–Methods ; Retinal Diseases–Methods ; Transduction, Genetic–Methods ; Recombinant Proteins
descriptionRetinal degenerative diseases such as retinal macular degeneration and retinitis pigmentosa constitute a broad group of diseases that all share one critical feature, the progressive apoptotic loss of cells in the retina. There is currently no effective treatment available by which the course of these disorders can be modified, and visual dysfunction often progresses to total blindness. Gene therapy represents an attractive approach to treating retinal degeneration because the eye is easily accessible and allows local application of therapeutic vectors with reduced risk of systemic effects. Furthermore, transgene expression within the retina and effects of treatments may be monitored by a variety of noninvasive examinations. An increasing number of strategies for molecular treatment of retinal disease rely on recombinant adeno-associated virus (rAAV) as a therapeutic gene delivery vector. Before rAAV-mediated gene therapy for retinal degeneration becomes a reality, there are a number of...
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titleRecombinant AAV-mediated gene transfer to the retina: gene therapy perspectives
descriptionRetinal degenerative diseases such as retinal macular degeneration and retinitis pigmentosa constitute a broad group of diseases that all share one critical feature, the progressive apoptotic loss of cells in the retina. There is currently no effective treatment available by which the course of these disorders can be modified, and visual dysfunction often progresses to total blindness. Gene therapy represents an attractive approach to treating retinal degeneration because the eye is easily accessible and allows local application of therapeutic vectors with reduced risk of systemic effects. Furthermore, transgene expression within the retina and effects of treatments may be monitored by a variety of noninvasive examinations. An increasing number of strategies for molecular treatment of retinal disease rely on recombinant adeno-associated virus (rAAV) as a therapeutic gene delivery vector. Before rAAV-mediated gene therapy for retinal degeneration becomes a reality, there are a number of...
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abstractRetinal degenerative diseases such as retinal macular degeneration and retinitis pigmentosa constitute a broad group of diseases that all share one critical feature, the progressive apoptotic loss of cells in the retina. There is currently no effective treatment available by which the course of these disorders can be modified, and visual dysfunction often progresses to total blindness. Gene therapy represents an attractive approach to treating retinal degeneration because the eye is easily accessible and allows local application of therapeutic vectors with reduced risk of systemic effects. Furthermore, transgene expression within the retina and effects of treatments may be monitored by a variety of noninvasive examinations. An increasing number of strategies for molecular treatment of retinal disease rely on recombinant adeno-associated virus (rAAV) as a therapeutic gene delivery vector. Before rAAV-mediated gene therapy for retinal degeneration becomes a reality, there are a number of...
copHoundmills
pubNature Publishing Group
doi10.1038/sj.gt.3302366
urlhttp://search.proquest.com/docview/218676687/
date2004-10-01