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. 1997 Sep;81(9):795–801. doi: 10.1136/bjo.81.9.795

Gene therapy for inherited retinal degeneration

R ALI 1, M REICHEL 1, D HUNT 1, S BHATTACHARYA 1
PMCID: PMC1722319  PMID: 9422936

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Figure 1  .

Figure 1  

Schematic of the mouse eye. Note that the lens is proportionally much larger in mice than in humans. The routes of intraocular injections are indicated.

Figure 2  .

Figure 2  

Blue X-gal staining indicates lacZ activity in the optic nerve and optic chiasma of a BALB/c mouse 3 days after subretinal injection of 2 µl suspension of replication competent HSV virus, BE8 (5 × 109 pfu/ml) in which the lacZ, driven by a CMV promoter, has been inserted into the non-essential Us5 gene.57

Figure 3  .

Figure 3  

Blue X-gal staining in the anterior chamber of adult BALB/c mouse after intracameral injection of 2 µl of adenovirus carrying a lacZ gene with nuclear localisation signal driven by a CMV promoter (AV.CMV.LacZnuc) at a titre of 1 × 109 pfu/ml. LacZ activity can be observed throughout the anterior segment including corneal endothelium, iris, and trabecular meshwork.

Figure 4  .

Figure 4  

Transduced corneal endothelium (A) and iris pigment epithelium (B) in adult BALB/c mouse after intracameral injection of 2 µl of AV.CMV.LacZnuc (1 × 109 pfu/ml). A 5 µm paraffin section counterstained with nuclear fast red (× 45).

Figure 5  .

Figure 5  

Transduced retinal pigment epithelium and occasional positive photoreceptor cell (arrow) in adult BALB/c mouse 2 weeks after subretinal injection of 2 µl of AV.CMV.LacZnuc (1 × 109 pfu/ml). A 5 µm paraffin section counterstained with nuclear fast red (× 66).

Figure 6  .

Figure 6  

Transduced photoreceptor cells in adult nude mouse 1 month after subretinal injection of 2 µl of AAV.CMV.LacZ (1 × 107 IU/ml). All the stained outer segments can be related to stained photoreceptor nuclei which is consistent with transduction of photoreceptor cells and subsequent transport of LacZ into the outer segments. A 5 µm paraffin section counterstained with nuclear fast red (×25).

Selected References

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