How could skipping exon 53 restore dystrophin expression

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Assignment:

1. There are approximately 140 million babies born each year worldwide. If this treatment is found to be successful, how many new people per year could see a benefit from treatment? How does this number affect the speed at which this research develops?

There are approximately 140 million babies born each year worldwide. If this treatment is found to be successful, how many new people per year could see a benefit from treatment? How does this number affect the speed at which this research develops?

2. How many nucleotides long is exon 53? How could skipping exon 53 restore dystrophin expression?

3. Why are phosphorodiamidate morpholino oligomers studied instead of RNA oligomers?

4. What is the model system they use for investigating this treatment? What are the benefits to using this system at this phase of the research?

Reference no: EM133635959

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