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Gene Therapy

I need help answering the following questions:

1. Description of gene therapy and how it works.

2. What are the principles that underlie gene therapy?

Please provide academic references to support your response.


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Gene therapy is a technique in which genetic material (mostly DNA) is introduced into the body to treat a disorder or even to prevent disease.

Gene therapy might work in three ways:

1) If production of a certain protein is deficient leading to a syndrome or disorder, a gene encoding that protein is introduced which then compensates to produce enough protein leading to a cure.

2) If a gene is mutated leading to a dysfunctional protein, the correct gene is introduced which compensates for lack of functional protein.

3) A gene encoding resistance to a particular disease may be introduced as a form of vaccination.

Based on what function the gene fulfills, there are two main types of gene therapy:

1) Somatic gene therapy

2) Germ line gene therapy

The main difference between them is that the former is not heritable (as the gene is introduced in adult differentiated cells) and the latter is heritable (as the gene is introduced in embryonic cells). Examples of somatic gene ...

Solution Summary

A description of gene therapy and how it works is provided. The principles that underlie gene therapy are given.