What are the potential risks of a treatment such as this

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Reference no: EM133619630

Questions:

1. What is the general method for screening an optimal exon-skipping sequence?

2. What is known about the duration of effect from this potential treatment?

3. Why are both RT-PCR and Western blots used in this study?

4. What efficiency of exon skipping is needed for therapeutic effects? Is this level achievable with this treatment?

5. What are the potential risks of a treatment such as this? How would those risks be studied and evaluated?

Reference no: EM133619630

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