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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


More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Clinical Neurosciences
Role:
Author
More by this author
Institution:
University of Oxford
Division:
MPLS
Department:
Engineering Science
Sub department:
Institute of Biomedical Engineering
Role:
Author
Journal:
Der Ophthalmologe : Zeitschrift der Deutschen Ophthalmologischen Gesellschaft
Volume:
109
Issue:
2
Pages:
121-128
Publication date:
2012-02-01
DOI:
EISSN:
1433-0423
ISSN:
0941-293X
Source identifiers:
312969
Language:
German
Keywords:
Pubs id:
pubs:312969
UUID:
uuid:13af9304-5fc0-43c7-8525-d9f5c1fd6d44
Local pid:
pubs:312969
Deposit date:
2012-12-19

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