Mucosal Delivery of RNAi Therapeutics - RNA Interference from Biology to Therapeutics - page 103

Formulation Route/animal Molecular target/model Effect (dosage) Ref./year

Chitosan Intratracheal B6;129P2-

RAGE tm1.1 mice

EGFP/EGFP transgenic mice 68% silencing of EGFP (3 × 0.26 m g) [ 13 ] /2010

Chitosan/imidazole-
PEG modi fi ed
chitosan

I.V./intranasal BALB/
c-C57BL/6 mice

GAPDH 40-50% silencing of GAPDH after I.V.

or intranasal administration
(1-3 × 0.5-1 mg)

[ 67 ] /2010

PEI Retroorbital injection—

C57BL/J mice

Nucleocapsid protein
in fl uenza A

10- to 1,000-fold reduction in viral titres
(1 × 120 m g)

[ 68 ] /2004

Fully deacetylated PEI Retroorbital injection—

C57BL/J mice

Nucleocapsid protein
in fl uenza A

94% drop in viral titre (1 × 120 m g) [ 69 ] /2005

PEG-PEI Intratracheal C57BL/6-Tg

(CAG-EGFP) 1 Osb/J mice

EGFP DsiRNA 42% knockdown of EGFP (1 × 50 m g) [ 57 ] /2009

Fatty acid modi fi ed
PEG-PEI

Intratracheal C57BL/6J-Tg mice EGFP 69% knockdown of EGFP (1 × 35 m g) [ 70 ] /2011

Lipid-based vectors
Oligofectamine/naked
siRNA

Intranasal /hydrodynamic
injection
BALB/cAnNCR mice

Nucleoprotein, acidic
polymerase/in fl uenza A

63-fold reduction of viral titres
( 1 ´ 50 mg + 20 m g)

[ 71 ] /2004

DharmaFECT Intratracheal C57BL/6 mice SPARC/bleomycin

induced lung fibrosis

58% reduced collagen content in lung (3 × 3 m g) [ 72 ] /2010

GL67 Intranasal BALB/c mice lacZ/ b -galactosidase 33% lower b-galactosidase mRNA levels

(1 × 40 m g)

Cholesterol/cell
penetrating peptides

Intratracheal BALB/c mice p38 MAP kinase 45% knockdown of p38 MAP kinase mRNA,

no change in protein levels (1 × 10 nmol)

[ 73 ] /2007

AtuPLEX I.V. C57BL/6N mice E-cadherin ~50% reduction of VE-cadherin mRNA

(4 × 50 m g)

[ 74 ] /2010

Infasurf Intranasal C57BL/6J mice GAPDH 50-67% lowered lung concentration of GAPDH

protein at 24 h and 7 days (1 × 10 m g)

[ 75 ] /2004