| 1 [ref. 84] |
|
|
|
In vitro cell assays |
99.1% cell uptake |
96–98% cell viability |
Deliver PNAs to primary CD34+ human hematopoietic stem cells |
| 2 [ref. 87] |
|
|
|
In vitro cell assays |
Confocal live-cell images confirm cell uptake |
No observed toxicity |
Block chemokine receptor 5 mRNA expression |
| 3 [ref. 88] |
|
|
|
In vitro and in vivo mouse model |
3-fold higher cell uptake with surface modification |
No observed toxicity |
Block micro-mRNA-155 expression and reduce tumor growth |
| 4 [ref. 89] |
|
|
|
In vivo mouse model |
Confocal live-cell images confirm cell uptake |
No observed toxicity |
Block micro-mRNA-155 expression and reduce tumor growth |
| 5 [ref. 8] |
|
|
|
In vivo mouse model |
Immune fluorescence microscopy confirms delivery into the cell nucleus |
No observed toxicity |
Editing of the mutated β-globin gene in hematopoietic stem cells |
| 6 [ref. 9 and 92] |
|
|
|
In vivo mouse model |
Confocal images of fetuses confirm in utero delivery after intra-amniotic administration |
No observed toxicity |
In utero editing of the mutated β-globin gene |
| 7 [ref. 100] |
|
|
|
In vitro cell assays |
Gal8 recruitment assay shows live-cell endosome-disruptive activity |
50%–90% cell viability |
Inhibit miRNA-122 expression |
| 8 [ref. 102] |
|
|
|
In vitro cell assays |
82% cell uptake |
∼100% cell viability |
Inhibit miRNA-155 expression and induce cell apoptosis |