Maximizing the potency of siRNA lipid nanoparticles for hepatic gene silencing in vivo.
Angew Chem Int Ed Engl
; 51(34): 8529-33, 2012 Aug 20.
Article
em En
| MEDLINE
| ID: mdl-22782619
ABSTRACT
Special (lipid) delivery The role of the ionizable lipid pK(a) in the in vivo delivery of siRNA by lipid nanoparticles has been studied with a large number of head group modifications to the lipids. A tight correlation between the lipid pK(a) value and silencing of the mouse FVII gene (FVII ED(50) ) was found, with an optimal pK(a) range of 6.2-6.5. The most potent cationic lipid from this study has ED(50) levels around 0.005 mg kg(-1) in mice and less than 0.03 mg kg(-1) in non-human primates.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Inativação Gênica
/
RNA Interferente Pequeno
/
Nanopartículas
/
Lipídeos
/
Fígado
Limite:
Animals
/
Female
/
Humans
Idioma:
En
Revista:
Angew Chem Int Ed Engl
Ano de publicação:
2012
Tipo de documento:
Article
País de afiliação:
Estados Unidos