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1. True or False: The goal of most therapeutic trials in spinal muscular atrophy is to increase the full-length SMN protein production from the SMN2 gene.Correct
2. Which of the following therapies is not utilized when trying to amplify SMN protein production?Correct
3. The first paper on gene replacement by intravenous delivery using scAAV9 was published in 2010 by Dr. Kevin Foust and colleagues. This study used a mouse model of SMA where the virus was injected by IV. On which day of the study was no effect shown?Correct