Preparation, Determination of Activity, and Biodistribution of Cholesterol-Containing Nuclease-Resistant siRNAs In Vivo.
Methods Mol Biol
; 2115: 57-77, 2020.
Article
en En
| MEDLINE
| ID: mdl-32006394
ABSTRACT
RNA interference (RNAi) is a powerful tool for suppressing gene expression associated with various diseases that are not amenable to treatment with low molecular weight drugs. Despite significant progress in this area, the potential for therapeutic use of RNAi in humans is limited due to the lack of efficient delivery systems. Bioconjugation is one of the most promising methods for delivering siRNA to cells and tissues, since conjugation of siRNA with molecules capable of penetrating cells through natural transport mechanisms can provide specificity of delivery without toxic effects and unwanted immunostimulation. Here we describe the design, preparation, and in vivo evaluation of cholesterol-containing siRNA conjugates able to accumulate in the tumor, penetrate into cells without a carrier, and suppress the expression of the target genes.
Palabras clave
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Colesterol
/
ARN Interferente Pequeño
Límite:
Animals
/
Humans
Idioma:
En
Revista:
Methods Mol Biol
Asunto de la revista:
BIOLOGIA MOLECULAR
Año:
2020
Tipo del documento:
Article
País de afiliación:
Rusia