Antisense oligonucleotides for target validation in the CNS.
Curr Opin Mol Ther
; 1(3): 336-43, 1999 Jun.
Article
em En
| MEDLINE
| ID: mdl-11713799
Although antisense oligonucleotides have been used in cell-based antisense experiments for nearly two decades, studies to investigate the function of CNS proteins in living animals were not successfully conducted until recently. Oligonucleotides are not transported across the blood-brain barrier to any appreciable extent. Consequently, these molecules need to be administered directly into the brain. Antisense approaches may be especially suited to investigation of CNS proteins. Due to their specificity, antisense sequences can more easily and selectively distinguish between closely related proteins, such as receptor subtypes, in contrast to the more traditional pharmacological agents such as small molecule ligands. This review discusses some unique technical aspects surrounding oligonucleotide delivery to the brain, and summarizes some of the more noteworthy applications of antisense tools to the study of CNS protein function during the past two years.
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Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Fármacos do Sistema Nervoso Central
/
Oligonucleotídeos Antissenso
/
Avaliação de Medicamentos
/
Proteínas do Tecido Nervoso
Tipo de estudo:
Prognostic_studies
Idioma:
En
Revista:
Curr Opin Mol Ther
Ano de publicação:
1999
Tipo de documento:
Article