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8. A type of gene therapy called RNA interference (RNAI) is being investigated to treat Huntington's disease. This disease is caused by a mutation in the DNA that results in the synthesis of an altered protein that leads to nervous system defects. To treat this disease, scientists synthesize short sequences of RNA (siRNA, or small interfering RNA) 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 in Problem 5. 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 the disease?

8. A type of gene therapy called RNA interference (RNAI) is being investigated to treat Huntington's disease. This disease is caused by a mutation in the DNA that results in the synthesis of an altered protein that leads to nervous system defects. To treat this disease, scientists synthesize short sequences of RNA (siRNA, or small interfering RNA) 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 in Problem 5. 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 the disease?

Biochemistry
Biochemistry
9th Edition
ISBN:9781319114671
Author:Lubert Stryer, Jeremy M. Berg, John L. Tymoczko, Gregory J. Gatto Jr.
Publisher:Lubert Stryer, Jeremy M. Berg, John L. Tymoczko, Gregory J. Gatto Jr.
Chapter1: Biochemistry: An Evolving Science
Section: Chapter Questions
Problem 1P
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5. Design a 10-bp primer that could be used to amplify the following sequence of DNA: 5'-AGTCGATCCCTGATCGTACGCTACGGTAACGT-3'
Transcribed Image Text:5. Design a 10-bp primer that could be used to amplify the following sequence of DNA: 5'-AGTCGATCCCTGATCGTACGCTACGGTAACGT-3'
8. A type of gene therapy called RNA interference (RNAi) is being investigated to treat Huntington's disease. This disease is caused by a mutation in the DNA that results in the synthesis of an altered protein that leads to nervous system defects. To treat this disease, scientists synthesize short sequences of RNA (siRNA, or small interfering RNA) 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 in Problem 5. 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 the disease?
Transcribed Image Text:8. A type of gene therapy called RNA interference (RNAi) is being investigated to treat Huntington's disease. This disease is caused by a mutation in the DNA that results in the synthesis of an altered protein that leads to nervous system defects. To treat this disease, scientists synthesize short sequences of RNA (siRNA, or small interfering RNA) 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 in Problem 5. 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 the disease?
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Small interfering RNA (siRNA): This is a type of RNA molecule that can trigger the degradation of a specific mRNA molecule, effectively silencing the gene that it codes for. This process is called RNA interference and is an important mechanism for regulating gene expression in cells.

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