Gene Therapy Approaches to Treat the Neurodegeneration and Visual Failure in Neuronal Ceroid Lipofuscinoses.
Adv Exp Med Biol
; 1074: 91-99, 2018.
Article
em En
| MEDLINE
| ID: mdl-29721932
ABSTRACT
Neuronal ceroid lipofuscinoses (NCLs) are a group of fatal, inherited lysosomal storage disorders mostly affecting the central nervous system of children. Symptoms include vision loss, seizures, motor deterioration and cognitive decline ultimately resulting in premature death. Studies in animal models showed that the diseases are amenable to gene supplementation therapies, and over the last decade, major advances have been made in the (pre)clinical development of these therapies. This mini-review summarises and discusses current gene therapy approaches for NCL targeting the brain and the eye.
Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Transtornos da Visão
/
Terapia Genética
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Degeneração Neural
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Lipofuscinoses Ceroides Neuronais
Tipo de estudo:
Etiology_studies
Limite:
Animals
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Child
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Humans
/
Infant
Idioma:
En
Revista:
Adv Exp Med Biol
Ano de publicação:
2018
Tipo de documento:
Article
País de afiliação:
Reino Unido