Intracerebral injections of DNA nanoparticles encoding for a therapeutic gene provide partial neuroprotection in an animal model of neurodegeneration.
Nanomedicine
; 13(7): 2209-2217, 2017 Oct.
Article
em En
| MEDLINE
| ID: mdl-28666950
ABSTRACT
This study reports proof of concept for administering compacted DNA nanoparticles (DNPs) intracerebrally as a means to protect against neurotoxin-induced neurodegeneration of dopamine (DA) neurons. In this study we used DNPs that encoded for human glial cell line-derived neurotrophic factor (hGDNF); GDNF is a potent neurotrophic factor for DA neurons. Intracerebral injections of DNPs into the striatum and/or substantia nigra were performed 1 week before treatment with a neurotoxin. We observed that the number of surviving DA cells, the density of DA fiber terminals and recovery of motor function were greater in animals injected with GDNF-encoding DNPs than in control animals receiving DNPs encoding for an inert reporter gene. The results of these studies are one of the first to demonstrate that a non-viral, synthetic nanoparticle can be used to deliver therapeutic genes to cells in the brain as a means to protect cells against neurotoxin-induced neurodegeneration.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
DNA
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Terapia Genética
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Técnicas de Transferência de Genes
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Fator Neurotrófico Derivado de Linhagem de Célula Glial
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Neurônios Dopaminérgicos
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Neuroproteção
Tipo de estudo:
Prognostic_studies
Limite:
Animals
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Humans
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Male
Idioma:
En
Revista:
Nanomedicine
Assunto da revista:
BIOTECNOLOGIA
Ano de publicação:
2017
Tipo de documento:
Article