- Tytuł:
- CEP290 gene transfer rescues Leber congenital amaurosis cellular phenotype.
- Autorzy:
- Źródło:
- Gene therapy [Gene Ther] 2014 Jul; Vol. 21 (7), pp. 662-72. Date of Electronic Publication: 2014 May 08.
- Typ publikacji:
- Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
- MeSH Terms:
-
Antigens, Neoplasm/*genetics
Induced Pluripotent Stem Cells/*transplantation
Leber Congenital Amaurosis/*therapy
Lentivirus/*genetics
Neoplasm Proteins/*genetics
Photoreceptor Cells/*metabolismRetina /*metabolism
Animals ; Antigens, Neoplasm/metabolism ; Cell Cycle Proteins ; Cells, Cultured ; Cilia/metabolism ; Cilia/pathology ; Cytoskeletal Proteins ; Disease Models, Animal ; Fibroblasts/metabolism ; Fibroblasts/pathology ; Genetic Vectors/pharmacology ; Humans ; Induced Pluripotent Stem Cells/metabolism ; Induced Pluripotent Stem Cells/pathology ; Leber Congenital Amaurosis/genetics ; Leber Congenital Amaurosis/pathology ; Mice ; Neoplasm Proteins/metabolism ;Retina /pathology ; Transduction, Genetic
Czasopismo naukowe