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Accession IconSRP125334

Triple vectors expand AAV transfer capacity in the retina

Organism Icon Homo sapiens
Sample Icon 12 Downloadable Samples
Technology Badge IconNextSeq 500

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Description
Maddalena et al. showed that the limited DNA transfer capacity (~4.7kb) of adeno associated viral (AAV) vectors can be expanded up to 14kb with triple AAV vectors for the efficient expression of the therapeutic CDH23 (10.1kb) and ALMS1 (12.5kb) genes. Overall design: cells infected with triple AAV vectors carrying 2 different transgenes in 3 biological replicates; RNA extracted from WT cells was used as control .
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12
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