The Best Treatment
Is gene therapy the answer to an untreatable form of macular degeneration?
Ruth Steer |
Most of the time, when you diagnose a patient with neovascular AMD, there are several management strategies that should help slow progression and retain vision – and there are many more in the research pipeline. But when a patient with Best disease (vitelliform macular degeneration) presents, it’s a very different story; currently, no medical or surgical management can help.
Now, a group from the University of Pennsylvania, Philadelphia, have unraveled some of the mysteries surrounding the pathology of Best disease – and developed a gene therapy that successfully treats the canine form (Figure 1) (1). Karina Guziewicz and Artur Cideciyan, lead authors of the study, tell us more about their research, and how they’re hoping to translate their work into the clinic...
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