In Gene Therapy for RP, LCA, the Earlier, the Better

Retinal Disease News

In Gene Therapy for RP, LCA, the Earlier, the Better
Vision Care And MaintenanceEyeGenomics
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A real-world study of an FDA-approved gene therapy for inherited retinal disease shows younger patients gain more visual improvements than adults, but doesn't rule out the procedure for the older people.

, has shown evidence in a real-world study from the United Kingdom that young children derive a greater improvement in vision than adults who undergo the procedure.

In his presentation, Jalil said full-field stimulus testing , which measures rod-mediated function of the photoreceptors, improved in all patients who had the assessments. The study population included two adults who had the gene-placement surgery in both eyes, and six children, four of whom had the procedure in both eyes.

Low-luminance visual acuity, which indicates vision in low-light situations, improved in the younger patients but not in the two adults, Jalil said.Treatment with voretigene neparvovec involves major vitreoretinal surgery in which a bleb, or bubble, is created under the retina and the gene vector is placed within it. ."But once the photoreceptors are irreversibly damaged, then it's difficult to get the visual function back.

However, he urged caution in treating adults:"You have to manage their expectations that they may see more in the dark, but there would not huge improvements if there is irreversible damage to the photoreceptors and the retina already."In his presentation, Jalil acknowledged the ongoing debate among retinal surgeons regarding whether the fovea should be detached or not during the procedure to place the vector.

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Vision Care And Maintenance Eye Genomics Genomic Medicine Surgery Vitreoretinal Surgery Vitreo-Retinal Surgeries Biologic Therapy Biologics Children Child Childhood Pediatrics Kids Gene Therapy Visual Acuity Retinitis Pigmentosa Uveitis Retina Rods UK Site Content United Kingdom Site Content United Kingdom UK Genetic Testing Macular Edema Macular Oedema

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