Genetic Therapy Modes Quiz

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Questions and Answers

What is the main strategy for targeting autosomal dominant genetic diseases using RNA therapies?

  • Activating the normal copy of the gene on the partner chromosome
  • Enhancing the expression of the defective gene
  • Silencing or limiting expression of the pathogenic gene by digesting its mRNA (correct)
  • Introducing a normal gene to complement the defective gene

Which type of RNAs is used to control gene function in RNA interference (RNAi)?

  • mRNAs
  • Antisense RNAs
  • siRNAs (correct)
  • RNA aptamers

What is the role of trans-splicing ribozyme in RNA therapeutics?

  • Targeting proteins
  • Encoding therapeutic proteins
  • Silencing pathogenic genes
  • Reprogramming genetic information (correct)

In treating diseases caused by recessive mutations, what is the primary aim of gene therapists?

<p>Complementing the defective gene (A)</p> Signup and view all the answers

Which type of RNA is used in targeting proteins like RNA aptamers in RNA therapies?

<p>mRNAs (C)</p> Signup and view all the answers

What distinguishes RNA interference (RNAi) molecules like siRNAs from other RNA therapeutics?

<p>They have characteristic 3′ overhangs. (A)</p> Signup and view all the answers

What is the role of siRNAs in the RNA-induced silencing complex (RISC)?

<p>Degrade target mRNA molecules (A)</p> Signup and view all the answers

How do aptamers function in cancer therapy?

<p>By selectively binding to specific targets (C)</p> Signup and view all the answers

What is the role of ribozymes in gene manipulation?

<p>Downregulating and repairing pathogenic genes (A)</p> Signup and view all the answers

Which ribozyme group is characterized by self-splicing abilities?

<p>Group I and group II intron ribozymes (A)</p> Signup and view all the answers

What is a unique feature of RNase P ribozyme discovered in E. coli?

<p>Independence from cellular proteins (D)</p> Signup and view all the answers

Which property distinguishes the hammerhead, hairpin, and HDV ribozyme motifs?

<p>Self-cleavage capability (C)</p> Signup and view all the answers

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