What is it?

It involves altering the genes inside your body’s cells in order to treat or stop disease. It’s applications are still being researched. It replaces faulty genes or adds new genes. It may treat many different types of diseases.$^2$

It is, however, illegal to insert genes into sex cells as the changes made would be inherited by the individual’s offspring. The individuals could end up passing their faulty alleles to the offspring, even if that individual is better off. $^2$

Example to Explain Gene Therapy

Take the disease that is a combined immunodeficiency that prevents bone marrow cells from producing a vital enzyme. This issue stems from a single gene. The lack of the vital enzyme results in a significant problem for the organism.$^1$

Steps to apply gene therapy to solve the problem:$^1$

1. Synthesize a RNA version of the normal allele for the gene of interest.
2. Insert it into the retrovirus. A retrovirus converts the RNA to DNA.
3. The retrovirus is then allowed to infect the the bone marrow cells.

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Basic Process For Gene Therapy

1. Doing research to find the gene involved in the genetic disorder.
2. Cutting out the normal allele. Special enzymes are used to do this.
3. Making many copies of the allele.
4. Insert copies of the normal alleles into the target cells of a person who has the genetic disorder, via liposome’s/plasmids or GM virus.
5. The normal gene is transcribed and translated.
6. A functioning protein is produced.

Pros and Cons

Types of Gene Therapy

There are two types of gene therapy. Ex vivo and in vivo.$^3$