Clinical trials are assessing a new treatment for age-related macular degeneration by the targeting vascular endothelial cell growth factor receptor (VEGFR) using RNAi technology. Which of the following statements most closely describes the characteristics of RNAi that make it useful as a therapeutic approach to disease?

Question

Quizplus · Accepted Answer

RNAi (RNA interference) works by specifically targeting and degrading mRNA molecules, preventing the translation of the target protein, in this case, VEGFR. The RNA inhibitory (RNAi) pathway in mammals involves the enzymatic processing of double-stranded RNA into small interfering RNAs (siRNAs) of approximately 22 to 25 nucleotides that may have evolved as a means to degrade the RNA genomes of RNA viruses such as retroviruses. Since RNAi reduces the level of the target mRNA the encoded protein is consequently reduced. Therefore, the RNAi technique can prove useful in treating diseases where expression of a mutant or aberrant protein is the cause of the symptoms such as the VEGFR in the progression of agerelated macular degeneration.

Clinical Trials Are Assessing a New Treatment for Age-Related Macular