Leber congenital amaurosis (LCA) has been the first inherited retinal disease with a gene therapy approved for clinical use. An article recently published in Acta Ophthalmologica reports on the short-term effects of this therapy.
The authors, a team led by Katarina Stingl at University of Tübingen, used an rtx1 adaptive optics camera to assess changes at the level of photoreceptor cells in the retina of a LCA patient after treatment.
“This case demonstrates a remarkable fast restoration of the central photoreceptors within only 5 weeks after treatment as documented by high-resolution imaging. The effect was still visible after 3 months.”
Changes in the photoreceptor mosaic were observed several weeks before an improvement in visual acuity could be measured.
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Article reference: Kortüm, F. C., et al. Short Term Morphological Rescue of the Fovea After Gene Therapy with Voretigene Neparvovec. Acta Ophthalmologica (2021) DOI:10.1111/aos.14990