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Question 1 of 3
1. Question
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.
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Question 2 of 3
2. Question
2. Which of the following therapies is not utilized when trying to amplify SMN protein production?
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Question 3 of 3
3. Question
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?
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