| Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain. |
| RNP-NC-no ligand |
| Editing efficiency calculated by counting the percent of NeuN+ neurons expressing tdTomato surrounding the injected area of the striatum |
| cerebral cortex |
| neuron |
| Editing Efficiency |
| 15000000947 |
|
| sNLS-SpCas9-sNLS |
| RNP-NC-no ligand |
| Nanoparticle |
| Ai14 gRNA |
|
|
| Mouse |
| B6;129S6-Gt(ROSA)26Sortm14(CAG-tdTomato)Hze/J |
|
| 18-19 weeks |
| Female |
|
| stereotaxic injection |
| intracerebral/intrastriatal |
| 30 pmol |
| once bilaterally |
| 0.2 μl/min |
| 1.5 microliters |
| 14 days |
| ribonucleoprotein (RNP) |
|
|
|
| Measured Values |
| Samples Size: | 4 |
| |
| Mean |
17.37 |
| 1 |
17.1 |
| 2 |
22.3 |
| 3 |
14.0 |
| 4 |
16.1 |
| |
| NC-No Ligand delivery of CRISPR RNP produces neuronal genome editing in animal UW2 |
|
|
Representative coronal brain images from an Ai14 reporter mouse intrastriatally injected with not decorated nanocages (NC-no ligand) carrying CRISPR Cas9/sgRNA ribonucleoprotein. The brain sections are shown in rostrocaudal (top to bottom) anatomical order. The colors correspond to tdTomato-expressing, genome-edited cells (red; white arrows pointing to edited area) with NeuN+ neurons (green), GFAP+ astrocytes (pink), and DAPI labelled nuclei (blue). |
| NC-No Ligand delivery of CRISPR RNP produces neuronal genome editing in animal UW3 |
|
|
Representative coronal brain images from an Ai14 reporter mouse intrastriatally injected with not decorated nanocages (NC-no ligand) carrying CRISPR Cas9/sgRNA ribonucleoprotein. The brain sections are shown in rostrocaudal (top to bottom) anatomical order. The colors correspond to tdTomato-expressing, genome-edited cells (red; white arrows pointing to edited area) with NeuN+ neurons (green), GFAP+ astrocytes (pink), and DAPI labelled nuclei (blue). |
|
