Gene therapy

Microperimetry as an Outcome Measure Improves Patient Eligibility and Efficacy Detection in RPGR Gene Therapy Trials

Microperimetry-based endpoints improved patient eligibility and detected treatment efficacy in a larger proportion of RPGR gene therapy trial participants than low luminance visual acuity.

Centripetal Retinal Degeneration Follows an Exponential Decay and May Be Reversed Following Gene Therapy

Fundus autofluorescence ring progression in rod-cone dystrophies followed a predictable exponential decay, with evidence of structural and functional reversal after RPGR gene therapy.