Why it is an effective mechanism to restore correct splicing

Reference no: EM13113755

One interesting idea for therapy has been explored in cell culture models. In this case the scientists expressed a specialized form of an snRNA that was normally found in the spliceosome. This specialized snRNA can hybridize with the aberrant splice site, resulting in an increase in the correct beta-globin mRNA. Why might this be an effective mechanism to restore correct splicing?

Reference no: EM13113755

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