Question

Phenylketonuria is a genetic disorder caused by a mutation in the gene for the enzyme phenylalanine hydroxylase, which eliminates its enzymatic activity. Could an antisense drug help individuals with this disorder?

a. Yes, if it binds to the mRNA of the phenylalanine hydroxylase gene and prevents its translation
b. Yes, if it is incorporated into the chromosome and prevents the expression of the phenylalanine hydroxylase gene
c. No, because mRNA does not persist in the cytoplasm of the cell
d. No, because blockage of phenylalanine hydroxylase gene expression will not remedy the original disorder

Answer 1

The correct answer is (a) Yes because antisense drugs, by binding to mRNA, prevent translation and can inhibit the synthesis of the defective enzyme associated with phenylketonuria (PKU).

Step-by-step explanation:

Antisense drugs are designed to bind to specific mRNA molecules, preventing their translation into proteins. In the case of phenylketonuria (PKU), caused by a mutation in the phenylalanine hydroxylase gene, an antisense drug could be developed to bind to the mRNA transcribed from this gene.

By doing so, it could inhibit the translation of the faulty mRNA, thus reducing the synthesis of the defective enzyme responsible for PKU. This approach aims to address the root cause of the disorder, providing a potential therapeutic strategy for individuals with PKU.