Prospects for gene therapy of inherited retinal disease
نویسندگان
چکیده
منابع مشابه
[Gene therapy for inherited retinal dystrophies].
The inherited retinal dystrophies comprise a large number of disorders characterized by a slow and progressive retinal degeneration. They are the result of mutations in genes that express in either the photoreceptor cells or the retinal pigment epithelium. The mode of inheritance can be autosomal dominant, autosomal recessive, X linked recessive, digenic or mitochondrial DNA inherited. At the m...
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Research into the molecular and genetic basis of disease is continually expanding and improving the prospects for rational treatments. Of these, gene therapy (here defined as the introduction of genetic material into human cells) may ultimately oVer the greatest scope. This article provides a perspective on the potential for gene therapy for treating inherited retinal degenerations, along with ...
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The photoreceptor cells of the retina are subject to a wide range of genetic diseases. This review summarizes current knowledge regarding an important group of retinal diseases caused by mutations in photoreceptor-enriched genes. In addition, progress toward treatment of a variety of these diseases in animal models via adeno-associated virus gene therapy is described. Although no human trials h...
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Substantial progress has been made over the last 20 years in understanding the basic biology and function of the normal nervous system, and in elucidating molecular and cellular mechanisms that underlie neurological disease. Together with these advances in understanding have come discoveries of novel genes and proteins, which collectively present an unprecedented opportunity to intervene in and...
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ژورنال
عنوان ژورنال: Eye
سال: 2009
ISSN: 0950-222X,1476-5454
DOI: 10.1038/eye.2008.412