Article
Targeting Nonsense Mutations in Diseases with Translational Read-Through-Inducing Drugs (TRIDs).
BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy - 1 Apr 2016
Nagel-Wolfrum Kerstin, Möller Fabian, Penner Inessa, Baasov Timor, Wolfrum Uwe
Abstract excerpt
In recent years, remarkable advances in the ability to diagnose genetic disorders have been made. The identification of disease-causing genes allows the development of gene-specific therapies with the ultimate goal to develop personalized medicines for each patient according to their own specific genetic defect. In-depth genotyping of many different genes has revealed that ~12% of inherited genetic disorders are...
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