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Journal article

[Gene therapy for retinal dystrophies].

Abstract:

Genetic mutations are the cause of inherited retinal dystrophies. The underlying genetic basis of these diseases suggests that a gene therapy approach is logical either to replace or reduce the expression of defective genes. The first proof-of-concept clinical studies in patients with Leber's congenital amaurosis have suggested that retinal gene therapy is safe and potentially effective, at least for specific disease entities. In contrast to pharmacological treatment gene therapy has the adva...

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Publication status:
Published

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Publisher copy:
10.1007/s00347-011-2453-3

Authors


Charbel Issa, P More by this author
More by this author
Institution:
University of Oxford
Department:
Oxford, MSD, Clinical Neuroscience
More by this author
Institution:
University of Oxford
Department:
Oxford, MSD, Clinical Neuroscience, Biomedical Research Centre
Journal:
Der Ophthalmologe : Zeitschrift der Deutschen Ophthalmologischen Gesellschaft
Volume:
109
Issue:
2
Pages:
121-128
Publication date:
2012-02-05
DOI:
EISSN:
1433-0423
ISSN:
0941-293X
URN:
uuid:13af9304-5fc0-43c7-8525-d9f5c1fd6d44
Source identifiers:
312969
Local pid:
pubs:312969

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