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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?

Complementing the defective gene Signup and view all the answers

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

mRNAs Signup and view all the answers

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

They have characteristic 3′ overhangs. Signup and view all the answers

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

Degrade target mRNA molecules Signup and view all the answers

How do aptamers function in cancer therapy?

By selectively binding to specific targets Signup and view all the answers

What is the role of ribozymes in gene manipulation?

Downregulating and repairing pathogenic genes Signup and view all the answers

Which ribozyme group is characterized by self-splicing abilities?

Group I and group II intron ribozymes Signup and view all the answers

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

Independence from cellular proteins Signup and view all the answers

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

Self-cleavage capability Signup and view all the answers