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

Gene therapy for inherited retinal degeneration

R R Ali  1 , M B Reichel, D M Hunt, S S Bhattacharya
Affiliations
Review

Gene therapy for inherited retinal degeneration

R R Ali et al. Br J Ophthalmol. 1997 Sep.
No abstract available

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Figures

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).
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