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Initial results of work on gene therapy for Best disease are promising

Best disease is a genetic condition that robs both children and young adults of their central vision. A new study conducted by the University of Pennsylvania has shown promising results in a possible gene therapy to treat Best disease, otherwise known as vitelliform macular dystrophy. Best disease is caused by an inherited mutation in the BEST1 gene and affects one in 10,000 people. Results of the study were published October 15 in PLOS ONE¬†and details the research team's initial findings using dogs as the initial study subjects. Researchers injected a harmless virus genetically modified to carry specific genetic material in 18 retinas in 12 dogs. Researchers then tracked the expression of the protein for up to six months and what they found was that the expression peaked four to six weeks after injection and remained stable for six months which indicated that the therapy would be long-lasting. Researchers believe that this therapy could have a great impact on vision impairment, color vision and issues with bright light.*  

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