Nl-Fr

View abstract

This abstract is assigned to session Poster session in O'Bistro
TitleVisual Function after Gene Therapy for RPE65-Related LCA
PurposeTo describe changes in visual function after gene therapy for RPE65-related Leber congenital amaurosis.
MethodsTwo 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).
ResultsBoth 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.
ConclusionGene 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.
Authors 1
Last nameUVIJLS
InitialsA
DepartmentDept Ophth, GUH
CityGhent
Authors 2
Last nameMaguire
InitialsAM
DepartmentFM Kirby Center for Mol Ophth, Penn
CityPhiladelphia
Authors 3
Last nameHigh
InitialsKA
DepartmentCtr Cell Mol Therapeutics, CHOP
CityPhiladelphia
Authors 4
Last nameWright
InitialsJF
DepartmentCtr Cell Mol Therapeutics, CHOP
CityPhiladelphia
Authors 5
Last namePierce
InitialsEA
DepartmentFM Kirby Center for Mol Ophth, Penn
CityPhiladelphia
Authors 6
Last nameDe Baere
InitialsE
DepartmentCtr for Med Genet, GUH
CityGhent
Authors 7
Last nameMarshall
InitialsKA
DepartmentCtr Cell Mol Therapeutics, CHOP
CityPhiladelphia
Authors 8
Last nameChung
InitialsDC
DepartmentFM Kirby Center for Mol Ophth, Penn
CityPhiladelphia
Authors 9
Last nameSun
InitialsJ
DepartmentCtr Cell Mol Therapeutics, CHOP
CityPhiladelphia
Authors 10
Last nameMcDonnell
InitialsJ
DepartmentCtr Cell Mol Therapeutics, CHOP
CityPhiladelphia
Authors 11
Last nameBennett
InitialsJ
DepartmentFM Kirby Center for Mol Ophth, Penn
CityPhiladelphia
Authors 12
Last nameLeroy
InitialsBP
DepartmentDept Ophth & Ctr Med Genet, GUH
CityGhent
top ^