Adeno-associated virus 8-mediated gene therapy for choroideremia: preclinical studies in in vitro and in vivo models. [electronic resource]
Producer: 20150330Description: 122-30 p. digitalISSN:- 1521-2254
- Adaptor Proteins, Signal Transducing -- genetics
- Animals
- Cell Line
- Cells, Cultured
- Choroideremia -- genetics
- Dependovirus -- genetics
- Disease Models, Animal
- Female
- Gene Expression
- Gene Order
- Genetic Therapy
- Genetic Vectors -- administration & dosage
- Heterozygote
- Humans
- Mice
- Mice, Knockout
- Retina -- metabolism
- Transduction, Genetic
- Transgenes
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Publication Type: Journal Article; Research Support, Non-U.S. Gov't
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