Gene replacement therapy rescues photoreceptor degeneration in a murine model of Leber congenital amaurosis lacking RPGRIP.
第一作者:
Basil S,Pawlyk
第一单位:
Berman-Gund Laboratory for the Study of Retinal Degenerations, Harvard Medical School, Massachusetts Eye and Ear Infirmary, 243 Charles Street, Boston, MA 02114, USA.
作者:
主题词
动物(Animals);盲(Blindness);细胞支架蛋白质类(Cytoskeletal Proteins);依赖病毒(Dependovirus);疾病模型, 动物(Disease Models, Animal);视网膜电描记术(Electroretinography);眼蛋白质类(Eye Proteins);荧光抗体技术, 间接(Fluorescent Antibody Technique, Indirect);基因表达调控(Gene Expression Regulation);遗传载体(Genetic Vectors);绿色荧光蛋白质类(Green Fluorescent Proteins);小鼠(Mice);小鼠, 基因敲除(Mice, Knockout);光感受器细胞, 脊椎动物(Photoreceptor Cells, Vertebrate);启动区, 遗传(Promoter Regions, Genetic);蛋白质类(Proteins);视网膜变性(Retinal Degeneration);视杆视蛋白(Rod Opsins);转染(Transfection)
DOI
10.1167/iovs.05-0371
PMID
16123399
发布时间
2020-12-09
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