A type of gene therapy called RNA interference (RNAi) is being investigated to treat Huntington’s disease. This disease is the result of a mutation in the DNA that results in the synthesis of a nervous system protein with an altered amino acid sequence. The mutated protein forms clumps, which cause nervous system defects. To treat this disease, scientists synthesize short sequences of RNA (siRNA, to be specific) that form base pairs with the mRNA that codes for the mutated protein. a. Design an siRNA that will interfere with the synthesis of the protein shown below (this is the coding DNA of the gene). b. Explain how the addition of the siRNA will prevent the synthesis of the protein. c. What are the difficulties that must be overcome in order for RNA interference to be an effective technique for treating disease?
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