Carried on chromosomes, our genes are known to be as both functional and basic physical units of heredity. Proteins are made by encoded instructions from genes. When genes alter, the proteins are unable to carry out their normal functions, thus resulting in genetic disorders. There are 3 different levels of categorizing genetic disorders:

• Level 1 Single Gene Disorders: When a mutation causes a protein product of a single gene to be altered or to be missing. 
• Level 2 Chromosome Abnormalities: This is when the entire chromosome or even large portions of them are either missing, duplicated, or altered. 
  
• Level 3 Multifactorial Disorders: This is when there are mutations in multiple genes, often due to environmental causes.

Because of a mutation, the new polypeptide that is created now has a different amino acid sequence. The effects of such change, can either be a small or a major issue. It all comes down to what function or role that the protein plays in the cell or organism that will determine how drastic the effects will be. 

(citation 2.5)

Genetic disorders can be distressing, and can make a huge impact on a person’s life. Since there is a wide variety of chromosomal disorders, they each affect a person in different ways. Some disorders can cause a person to act obsessive and/or compulsive.Some can even lead to other health problems which affects the ability of a person to partake in everyday activities. Some chromosomal disorders can also affect a person’s capability of having children, whereas others can also create mental and/or physical disabilities.(citation 1.6)

What can be done about genetic disorders that affect the lives of numerous amounts of people in the world? This is where gene therapy comes in and can change the lives of many for a better future!

(citation 1.6)

The picture on the left shows how a chromosome chart of a normal person without genetic defects would look like. On the right, the chart shows chromosomes of a person who has Trisomy-Down Syndrome. As you can see, there is an extra chromosome on 21. Differences like these can drastically change a person's life. 

(citation 2.6) (citation 2.7)

Researchers are investigating many different ways to correct defective genes in order to cure that disease, or to even help a person’s body ( who is diagnosed with the disease) fight it. Some of the ways are:

• By replacing the missing or altered gene: This approach is currently the most commonly applied. A normal gene is inserted into “a nonspecific location within the genome to replace a nonfunctional gene” (citation 1.4)

• Another way is to change the regulation of a gene: Healthy genes which help to prevent diseases can be turned on so that they can restrain the disease, whereas mutated genes, that actually cause the disease, can be turned off so that they no longer elevate the disease.

• By making the immune system recognize diseased cells: Sometimes, our immune systems do not recognize the diseased cells as intruders in our body. By the use of gene therapy, doctors can introduce the mutated cells with the genes in which they could be recognized by our immune systems. Another approach is by enhancing immune cells so that it would be easier for them to identify mutated cells that are threatful to our bodies.

  (citation 1.7)

Click on this video to find out more about Gene Therapy from Dr. Ann Stapleton, an assistant professor at University of North Carolina at Wilmington!

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