A 47-year-old man is being examined by his personal physician because of a progressive dementia, evolving involuntary movements, and changes to his affect. History indicates that he noticed frequent uncontrolled spasmodic twitching of his fingers beginning about 1 year ago. Physical examination indicates clumsiness, hyperreflexia, and eye movement disturbances. You make a diagnosis and suggest that the patient is an ideal candidate for a new form of therapy. Which of the following techniques is most likely associated with the therapy for the suspected disease?

Question

Quizplus · Accepted Answer

RNAi is a technique that can be used to silence genes. It is a promising new therapy for a variety of diseases, including Huntington's disease.  The patient is suffering from Huntington disease which results from a trinucleotide expansion in the gene encoding the huntingtin protein. Since this is an abnormal protein it is an amenable target for RNAi therapy. RNAi involves a mechanism of RNA-directed regulation of gene expression principally at the level of the mRNA product of the targeted gene. Interference can involve mRNA degradation or a block to translation or both.

A 47-Year-Old Man Is Being Examined by His Personal Physician