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INDOLE-DERIVED COMPOUNDS INHIBITOR OF THE NONSENSE-MEDIATED mRNA DECAY (NMD) FOR THE TREATMENT OF GENETIC DISEASES |
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Principal Investigator: F. Lejeune
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TECHNICAL DESCRIPTION
This invention describes new molecules derived from Indole with the property to inhibit specifically the nonsense-mediated mRNA decay (NMD) process. This mRNA degradation process identified in all eukaryotes organism play an important role in the quality control of mRNA. This process takes place on mRNA with an early stop codon which is responsible of numerous genetic diseases.
The invention relates to the use of these derivatives for the production of drugs for the treatment of genetic diseases related to the absence of a gene expression due to NMD.
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