THE BELGIAN OPHTHALMIC ASSOCIATIONS
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| Titre | Visual Function after Gene Therapy for RPE65-Related LCA |
| But | To describe changes in visual function after gene therapy for RPE65-related Leber congenital amaurosis. |
| Méthodes | Two male Belgian patients of 9 and 10 years old at the first treatment underwent an extensive ophthalmological work-up, including psychophysical testing. Here, we focused on best-corrected visual acuity (BCVA), Goldmann visual fields (GVF) and a Low Vision Panel D-15 colour vision test. Both patients were treated with consecutive vitrectomies with a subretinal injection of AAV2-hRPE65v2 ((4,8x1010 and 1,5x1010 vector genomes in 1st and 2nd eyes respectively). |
| Résultats | Both patients showed a significant bilateral improvement of BCVA after treatment of the first eye, but BCVA did not significantly change after treatment of the second eye. A clear improvement in GVF of both eyes was noted after treatment of the first eye, with further improvement of both GVFs, after the second eye was treated. Colour vision did not improve significantly following gene therapy. |
| Conclusion | Gene therapy with subretinal injection of AAV2-hRPE65v2 is a safe procedure, but also significantly improves retinal sensitivity and consequently visual function. This is most clearly illustrated by the improved GVF. |
Auteur 1
| Nom | UVIJLS |
| Initiales | A |
| Institut | Dept Ophth, GUH |
| Ville | Ghent |
Auteur 2
| Nom | Maguire |
| Initiales | AM |
| Institut | FM Kirby Center for Mol Ophth, Penn |
| Ville | Philadelphia |
Auteur 3
| Nom | High |
| Initiales | KA |
| Institut | Ctr Cell Mol Therapeutics, CHOP |
| Ville | Philadelphia |
Auteur 4
| Nom | Wright |
| Initiales | JF |
| Institut | Ctr Cell Mol Therapeutics, CHOP |
| Ville | Philadelphia |
Auteur 5
| Nom | Pierce |
| Initiales | EA |
| Institut | FM Kirby Center for Mol Ophth, Penn |
| Ville | Philadelphia |
Auteur 6
| Nom | De Baere |
| Initiales | E |
| Institut | Ctr for Med Genet, GUH |
| Ville | Ghent |
Auteur 7
| Nom | Marshall |
| Initiales | KA |
| Institut | Ctr Cell Mol Therapeutics, CHOP |
| Ville | Philadelphia |
Auteur 8
| Nom | Chung |
| Initiales | DC |
| Institut | FM Kirby Center for Mol Ophth, Penn |
| Ville | Philadelphia |
Auteur 9
| Nom | Sun |
| Initiales | J |
| Institut | Ctr Cell Mol Therapeutics, CHOP |
| Ville | Philadelphia |
Auteur 10
| Nom | McDonnell |
| Initiales | J |
| Institut | Ctr Cell Mol Therapeutics, CHOP |
| Ville | Philadelphia |
Auteur 11
| Nom | Bennett |
| Initiales | J |
| Institut | FM Kirby Center for Mol Ophth, Penn |
| Ville | Philadelphia |
Auteur 12
| Nom | Leroy |
| Initiales | BP |
| Institut | Dept Ophth & Ctr Med Genet, GUH |
| Ville | Ghent |