refine.bio helps you build ready-to-use datasets with normalized transcriptome data from all of the world’s genetic databases.
SRP125334
Triple vectors expand AAV transfer capacity in the retina
Homo sapiens
12 Downloadable Samples
NextSeq 500
Submitter Supplied Information
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 .