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Gene expression is the required process in eukaryotes in which DNA is transcribed into pre-mRNA, spliced to produce "mature" mRNA and translated into proteins. This process is highly regulated and requires high fidelity. Inaccuracies in splicing events are receiving more attention as there is the finding that nearly 59% of all genes express alternative forms of mRNA. Spinal Muscular Atrophy (SMA) the disease that this project studies is not caused by alternative RNA splicing, but rather a duplicate gene product is skewed due to an inherent alternative splicing event. To expand on the knowledge of how a mirror copy of the causative gene (SMN2) can be regulated, we developed multiple types of small RNA molecules to manipulate the alternative splicing process. This manipulation had an ultimate goal of increasing the inclusion of a required exon, exon 7. It is known in SMA if there is an increase in full length expression there is a correlative decrease in disease severity. To better understand how aberrant RNA processing events occur and how we could manipulate these signals, we identified two trans elements associated with survival motor neuron (SMN) pre-mRNA splicing, and targeted

Gene therapy in spinal muscular atrophy : RNA-based strategies to modulate the pre-mRNA splicing of survival motor neuron