| abstract |
The invention relates to new compounds and pharmaceutical composition comprising such compounds, which are useful to treat diseases related to the process of splicing pre-messenger RNAs in the cell. Said disease can be Frasier syndrome, frontotemporal dementia related to chromosome 17, Leigh syndrome, atypical cystic fibrosis, certain neuropathologies including Alzheimer's related to a mutation of the Tau protein, amyotrophy which affects the Survival Motor Neurons (SMN) gene, depression related to dysregulation of serotonin splicing, or certain metastatic cancers in which the overall splicing process is affected. Said disease related to the process of splicing pre-messenger RNAs in the cell can appear with aging or can be of viral origin. can be associated with gene mutations and can be treated by exon skipping. The compounds can also be used to treat a disease associated with gene mutations, which disease can be treated by exon skipping. |