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Deze abstract is toegekend aan sessie Poster session in O'Bistro

Titel

Visual Function after Gene Therapy for RPE65-Related LCA

Doel	To describe changes in visual function after gene therapy for RPE65-related Leber congenital amaurosis.
Methodes	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).
Resultaten	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.
Conclusie	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

Naam	UVIJLS
Initialen	A
Instituut	Dept Ophth, GUH
Stad	Ghent

Auteur 2

Naam	Maguire
Initialen	AM
Instituut	FM Kirby Center for Mol Ophth, Penn
Stad	Philadelphia

Auteur 3

Naam	High
Initialen	KA
Instituut	Ctr Cell Mol Therapeutics, CHOP
Stad	Philadelphia

Auteur 4

Naam	Wright
Initialen	JF
Instituut	Ctr Cell Mol Therapeutics, CHOP
Stad	Philadelphia

Auteur 5

Naam	Pierce
Initialen	EA
Instituut	FM Kirby Center for Mol Ophth, Penn
Stad	Philadelphia

Auteur 6

Naam	De Baere
Initialen	E
Instituut	Ctr for Med Genet, GUH
Stad	Ghent

Auteur 7

Naam	Marshall
Initialen	KA
Instituut	Ctr Cell Mol Therapeutics, CHOP
Stad	Philadelphia

Auteur 8

Naam	Chung
Initialen	DC
Instituut	FM Kirby Center for Mol Ophth, Penn
Stad	Philadelphia

Auteur 9

Naam	Sun
Initialen	J
Instituut	Ctr Cell Mol Therapeutics, CHOP
Stad	Philadelphia

Auteur 10

Naam	McDonnell
Initialen	J
Instituut	Ctr Cell Mol Therapeutics, CHOP
Stad	Philadelphia

Auteur 11

Naam	Bennett
Initialen	J
Instituut	FM Kirby Center for Mol Ophth, Penn
Stad	Philadelphia

Auteur 12

Naam	Leroy
Initialen	BP
Instituut	Dept Ophth & Ctr Med Genet, GUH
Stad	Ghent