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Article

The Role of the Human Visual Cortex in Assessment of the Long-Term Durability of Retinal Gene Therapy in Follow-on RPE65 Clinical Trial Patients.

Ophthalmology - 1 Jun 2017

Ashtari Manzar, Nikonova Elena S, Marshall Kathleen A, Young Gloria J, Aravand Puya, Pan Wei, Ying Gui-Shuang, Willett Aimee E, Mahmoudian Mani, Maguire Albert M, Bennett Jean

Abstract excerpt

PURPOSE: Gene therapy (GT) has offered immense hope to individuals who are visually impaired because of RPE65 mutations. Although GT has shown great success in clinical trials enrolling these individuals, evidence for stability and durability of this treatment over time is still unknown. Herein we explored the value of functional magnetic resonance imaging (fMRI) as an objective measure to assess independently...

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Source

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Identifiers

PMID: 28237426
DOI: 10.1016/j.ophtha.2017.01.029
PMCID: PMC5805133

Genes

RPE65

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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