Cystic fibrosis is a genetic disease that results from a defective CFTR protein that alters ion flow through the cell membrane such that water does not cross the cell membrane. Gene therapy is being used to attempt to help cystic fibrosis patients. Which of the following steps is not needed to develop a gene therapy treatment for cystic fibrosis?
A) Clone the normal-functioning CFTR gene and make an RNA version of the gene.
B) Make antibodies to the defective CFTR protein to enhance the patient's immune system.
C) Remove cells from a patient and infect them with the recombinant virus.
D) Insert the RNA version of the CFTR gene into a virus.

Question

Quizplus · Accepted Answer

Making antibodies to the defective CFTR protein is not necessary for the development of a gene therapy treatment for cystic fibrosis. The focus of gene therapy for cystic fibrosis is on replacing or repairing the faulty CFTR gene, not enhancing the immune system's response to the defective protein. The other steps listed (cloning the normal-functioning CFTR gene and making an RNA version of it, inserting the RNA into a virus, and infecting the patient's cells with the recombinant virus) are all essential steps in the process of developing a gene therapy treatment for cystic fibrosis.

Cystic Fibrosis Is a Genetic Disease That Results from a Defective