Toward a therapeutic strategy for polyalanine expansions disorders: In vivo and in vitro models for drugs analysis

Here we report a list of drugs whose effects have been assayed both in in vitro and in vivo models of polyalanine disorders, such as the oculopharyingeal muscular dystrophy, congenital central hypoventilation syndrome, synpolydactyly and in cell and animal models carrying specific artificial mutations. In particular, we have reviewed, for each polyalanine mutant protein, the molecules tested, cellular models under investigation, drugs effects on aggregation and underlying mechanisms.