Which is NOT used in gene therapy?

Appeared in: UP NHM CHO 7 Sept 2022 (Shift-2)

Explanation

Gene therapy's fundamental goal is to correct genetic defects to treat or cure a disease.
A mutated gene is, by definition, faulty and produces a non-functional or harmful protein.
Activating a mutated gene would increase the production of this faulty protein, which would worsen the patient's condition.
This action is directly contrary to the therapeutic purpose of gene therapy.

Option B: This is a valid and common gene therapy strategy known as gene silencing or gene knockdown.
Option C: This is a primary strategy in gene therapy, often called gene augmentation.
Option D: This is a valid, though more complex, strategy often referred to as gene editing or gene correction.

Clinical Relevance

Nursing practice connection: Knowing Principles and strategies of gene therapy helps nurses interpret findings accurately and avoid errors in routine assessment, medication administration, and patient teaching.
Nurses play a crucial role in caring for patients undergoing gene therapy, which includes patient education about the procedure, managing potential side effects (like immune reactions to the vector), and long-term monitoring.
Gene therapy is no longer theoretical; approved treatments exist for conditions like spinal muscular atrophy (Zolgensma®), sickle cell disease (Casgevy®), and certain types of hemophilia and inherited blindness.
What if the disease is a dominant negative disorder (e.g., Huntington's disease)? In that case, simply 'introducing a new gene' (gene augmentation) would be insufficient because the faulty protein from the mutated gene would still be present and cause harm. The necessary approach would be to 'inactivate the mutated gene' to stop the production of the toxic protein.

How to Approach the Question

First, identify the negative keyword 'NOT' in the question stem. This signals that you must find the option that is an invalid or incorrect statement.
Recall the fundamental purpose of gene therapy: to fix a genetic problem that causes disease.
Evaluate each option against this core purpose.
Consider 'Inactivating a mutated gene.' If a gene makes a harmful product, turning it off would be therapeutic. This is a valid strategy.
Consider 'Introducing a new gene.' If a person lacks a working gene, adding one would be therapeutic. This is a valid strategy.
Consider 'Replacing a disease-causing gene.' Directly fixing the error is the ultimate goal. This is a valid strategy.

Concept Tested & Keywords

QfHIPQi5mySNQ4xTd-v4nb

E6 Medicine Harrison 22e Part 2 p. 1727-1729

E6 Harper's Illustrated Biochemistry2023 (pp 26-793 of 813) p. 438-440

Practise the full UP NHM CHO 7 Sept 2022 (Shift-2)

Attempt every question from this paper in a timed mock, then review the full solution for each one.