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Team takes first step toward macular dystrophy gene therapy

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Posted October 16, 2013

Vitelliform macular dystrophy, also known as Best disease, is one of a group of vision-robbing conditions called bestrophinopathies that affect children and young adults. Caused by inherited mutations in the BEST1 gene, these diseases cause severe declines in central vision as patients age.

Penn researchers take first step toward a macular dystrophy gene therapy
A photomicrograph collage shows the subretinal space of a vision-impaired dog with a gene therapy vector delivering a “healthy” copy of the affected gene. Credit: Confocal photomicrograph: Karina E. Guziewicz, Art: Mary Leonard

With a new study, University of Pennsylvania researchers report “encouraging” findings that mark the first clear step in developing a gene therapy that could prevent vision loss or event restore vision in individuals suffering from these conditions.

The research, conducted in dogs, which can naturally develop a disease similar to Best disease, was led by Karina E. Guziewicz and Gustavo D. Aguirre of Penn’s School of Veterinary Medicine. Penn Vet collaborators included Barbara Zangerl, Andras M. Komaromy, Simone Iwabe and William A. Beltran. The Penn team worked with University of Florida investigators Vincent A. Chiodo, Sanford L. Boye and William W. Hauswirth. They reported their findings in the journal PLOS ONE.

Read more at: MedicalXpress

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