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Alternative Splicing Modification as a Treatment for Genetic Disorders

Martinez Contreras Rebeca and Martinez Montiel Nancy

Alternative splicing is a co-transcriptional mechanism that regulates eukaryotic gene expression affecting the majority of human genes. In this mechanism, different sequences can be identified and removed from the pre-mRNA. Using alternatives splicing, multiple mRNA combinations of joined sequences can be produced from a single gene, increasing the coding potential of the genome. Malfunctions of alternative splicing events can affect the natural expression of different transcripts. Several strategies have been developed to regulate alternative splicing and the mechanisms underlying the functional and physiological implications of these tools are diverse. Collectively, these strategies are intended to improve the treatment of human genetic diseases.