Question

In the context of Huntington's disease, the siRNA inhibition of expression of mutated HTT gene will:

a) Aggravate the pathology
b) Have no effect on the pathology
c) Partially alleviate the pathology
d) Completely alleviate the pathology

Answer 1

Using siRNA to inhibit the mutated HTT gene in Huntington's disease can partially alleviate symptoms by reducing the production of the abnormal huntingtin protein.

Step-by-step explanation:

In the context of Huntington's disease, the use of siRNA (small interfering RNA) to inhibit the expression of the mutated HTT gene has the potential to partially alleviate the pathology associated with the disease. This therapeutic approach aims to reduce the production of the abnormal huntingtin protein, which leads to the death of brain cells, by targeting and degrading the messenger RNA (mRNA) before it can be translated into protein. Because the underlying genetic mutation is still present and because siRNA therapy may not fully eliminate the production of the mutant protein, this treatment might not completely alleviate the pathology, but it can certainly reduce the severity of the symptoms by decreasing the amount of harmful protein produced. Thus, the most accurate answer is (c) Partially alleviate the pathology.