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INDOLE-DERIVED COMPOUNDS FOR THE TREATMENT OF DISEASES RELATED TO THE SPLICING PROCESS |
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Principal Investigator: J. TAZI
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TECHNICAL DESCRIPTION
Human genome contains about 35 000 genes but is able to produce about 250 000 proteins. This diversity is mainly due to alternative splicing which allows the modulation of the exon contents of mRNAs. The prevalence of alternative splicing as a general mechanism to control gene expression makes it a privileged target for alterations leading to pathologies. Along this line, up to 50% of all point mutations responsible for human diseases manifest themselves as splicing defects.
The inventors have discovered small molecules, derived to indole compounds that target selectively key components of alternative splicing machinery.
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