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Research Field Cell & gene therapy, Neuroscience

The Best Treatment

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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About the Author

Ruth Steer

Following my journey through academia, I entered the world of scientific writing and never looked back. After several years of working as a medical writer – where I developed a wide range of skills in healthcare publications and communications – I took the opportunity to stretch my creative and journalistic muscles and joined Texere Publishing. Working as Editor on The Ophthalmologist allows me to nurture my skills in scientific writing – and explore the dynamic world of ophthalmology – all within an innovative and exciting company.

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