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The rapid expansion of our knowledge of the human genome led to the promise of new drugs and therapeutics approaches. If the gene therapy is still in its infancy because of the difficulties to successfully deliver the right dose of the gene-drug to the right cell, alternative approaches have emerged.
Among them, the exon-skipping approach aims to use small molecules (modified oligonucleotides) to mask specific signals (splicing signals) to the cellular machinery to induce an exon-skipping. This approach can restore a fully normal transcript (skipping of a cryptic exon generated by a deep intronic mutation), or an internally deleted transcript that conserves enough activity. Alternatively, this approach can be used to silence a transcript.
The exon skipping approach has been successfully used in various contexts such as Duchenne Muscular Dystrophy or Dysferlinopathies. Because the selection of the antisense oligonucleotides (AON) is difficult, in collaboration with the Aix Marseille University, the GenOmnis company has designed a dedicated system to select the most relevant AON of various sizes (15 to 40 nt) to induce exon skipping of all human exons.
A second approach is the nonsense read through. Its principle is to interact with the ribosome in order to make it recognize a premature nonsense codon as a normal codon and therefore to incorporate an amino acid instead of stopping the protein elongation. Various molecules are today able to promote this mechanism. Nevertheless, all amino acid residues are not equal for nonsense read through. in collaboration with the Aix Marseille University, the GenOmnis company has designed a dedicated system to select nonsense mutations that will produce a functional protein after semi-random incorporation of an amino-acid at the residue of the nonsense mutation.
Other specific systems can be developed on-demand, to do so, please contact us.