Answered

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For each of the following genetic conditions, state which of the three types of gene therapy we covered (gene introduction, gene silencing, or gene editing) would be best suited as a treatment and briefly explain your rationale.
a) An autosomal dominant disease of the immune system that is caused by a point mutation in a single gene in white blood cells.
b) An autosomal recessive disease of the lungs caused by a lack of production of a protein (surfactant) that helps keep the surface of the lung smooth and moist.
c) An autosomal dominant neuromuscular disease caused by the production of a protein in childhood that should only be produced during fetal development.

Sagot :

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Answer:

a) gene editing

b) gene introduction

c) gene silencing

Explanation:

1.  In this scenario, the disease is being caused by a problem at a singular point of the DNA. By changing this one point through gene editing, all of the white blood cells will be corrected and start working correctly.

2. Since not enough protein is not being produced because of the lack of a specific DNA code to produce more, this can be resolved by adding in the code to produce the protein through gene introduction.

3. The gene that causes production of a problematic protein can be silenced to prevent it from being translated again, therefore stopping the protein from being produced.

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