ALMS is caused by mutations in ALMS1 (CDS, 12.5 kb), which encodes for a ciliary/centrosomal protein thought to play a key role in transport along the PR axoneme.34, 35, 36, 37, 38 Triple AAV vectors have previously been exploited to reconstruct full-length dystrophin (CDS, 11.1 kb) in the muscle of dystrophic mice.39, 40 Low levels of full-length dystrophin expression were obtained by both TS40 and hybrid systems.39 This evidence concerns the gene ALMS1 and Alstrom syndrome.