Gene Therapy in Neovascular Age-related Macular Degeneration: Three-Year Follow-up of a Phase 1 Randomized Dose Escalation Trial.

@article{Constable2017GeneTI,
  title={Gene Therapy in Neovascular Age-related Macular Degeneration: Three-Year Follow-up of a Phase 1 Randomized Dose Escalation Trial.},
  author={Ian Jeffery Constable and C. M. Lai and Aaron Len Magno and Martyn A French and Samuel B. Barone and Steven D Schwartz and Mark Scott Blumenkranz and Mariapia A Degli-Esposti and Elizabeth Piroska Rakoczy},
  journal={American journal of ophthalmology},
  year={2017},
  volume={177},
  pages={150-158}
}
PURPOSE To assess the safety of rAAV.sFlt-1 subretinal injection in neovascular age-related macular degeneration (wet AMD) over 36 months. DESIGN Phase 1 dose escalation trial. METHODS Eight subjects with advanced, treatment-experienced wet AMD were randomly assigned (3:1) to treatment and non-gene therapy control groups. Eligible subjects were ≥65 years, had wet AMD, and had best-corrected visual acuity (BCVA) 10/200 to 20/80 in the study eye and 20/200 or better in the other eye. Three of… CONTINUE READING
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