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An AAV Dual Vector Strategy Ameliorates the Stargardt Phenotype in Adult Abca4 −/− Mice

1 Nov 2018

Abstract excerpt

The recent approval in the United States of the first adeno-associated viral (AAV) vector for the treatment of an inherited retinal degeneration validates this approach for the treatment of many other diseases. A major limiting factor continues to be the size restriction of the AAV transgene at under 5 kb. Stargardt disease is the most prevalent form of recessively inherited blindness and is caused by mutations...

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PMID: 30381971
DOI: 10.1089/hum.2018.156

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