Pluto Bioinformatics

GSE107173: Triple vectors expand AAV transfer capacity in the retina

Bulk RNA sequencing

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. SOURCE: Margherita Mutarelli (mutarelli@tigem.it) - TIGEM