Progress in research on pathogenic genes and gene therapy for inherited retinal diseases / 中华医学遗传学杂志
Chinese Journal of Medical Genetics
;
(6): 118-123, 2017.
Artigo
em Chinês
| WPRIM
| ID: wpr-335169
ABSTRACT
Inherited retinal diseases (IRDs), including retinitis pigmentosa, Usher syndrome, Cone-Rod degenerations, inherited macular dystrophy, Leber's congenital amaurosis, Leber's hereditary optic neuropathy are the most common and severe types of hereditary ocular diseases. So far more than 200 pathogenic genes have been identified. With the growing knowledge of the genetics and mechanisms of IRDs, a number of gene therapeutic strategies have been developed in the laboratory or even entered clinical trials. Here the progress of IRD research on the pathogenic genes and therapeutic strategies, particularly gene therapy, are reviewed.
Texto completo:
DisponíveL
Índice:
WPRIM (Pacífico Ocidental)
Assunto principal:
Doenças Retinianas
/
Terapêutica
/
Terapia Genética
/
Ensaios Clínicos como Assunto
/
Resultado do Tratamento
/
Predisposição Genética para Doença
/
Pesquisa Biomédica
/
Genética
/
Métodos
/
Mutação
Limite:
Humanos
Idioma:
Chinês
Revista:
Chinese Journal of Medical Genetics
Ano de publicação:
2017
Tipo de documento:
Artigo
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