Gene+Therapy

= Gene Therapy =

How is genetic engineering used within gene therapy?

1) __What is genetic engineering?:__ Genetic engineering is the process of transferring specific genes from the chromosome of one organism and transplanting them into the chromosome of another organism in such a way that they become a reproductive part of the new organism. (1

(2

2) __What is gene therapy?__ The insertion of usually genetically altered genes into cells especially to replace defective genes in the treatment of genetic disorders or to provide a specialized disease-fighting function (3

(4

Going back to how is genetic engineering used within gene therapy, we can see how it is applied towards the treatment of genetic diseases. Since the genes in all our cells contain the code for proteins that have the function and structure to all our organs and tissues, this gives information towards finding treatments towards thise diseases. (5 __How is it done?:__ In order to cure a genetic illness, the gene must be replaced. You do so by inserting the normal gene and replacing the irregular one. If you are able to find the gene in an early stage, this will cause the normal gene to form part of all the tissues. (5

1)[|Infoplease]2010- 11 -16 2)[|geneticpeople] 2010 - 11- 16 3)[|Britannica] 2010 - 11- 16 4)[|mrheath] 2010 - 11- 16

5)[|humgene]2010 - 11 - 17

Features:
 * Gene therapy can make a set of instructions of cells.
 * When a gene has a malformation, it can create many diseases. By gene therapy, doctors can fix these mistakes through the cells.
 * The investigation and use of gene therapy is just starting, but with more years of experience and investigation on this topic, it could be possible that it could cure many incurable diseases like cardiovascular disease, cancer, hemophilia, cystic fibrosis, etc.
 * One way of doing gene therapy is by taking out immune cells from the persons body and adding the ADA gene. After this, they place the cells back to the body. By doing this, the ADA gene makes the cells to create a normal quantity of the enzyme that is defective or simply not there and the immune system would be good as new.
 * However gene therapy can also help cure other diseases that have nothing to do with genetic; that have other repercussions.
 * You can insert genes that have been changed or are completely new and make the cells concentrate on extremely different tasks.
 * For example, inserting genes to cancer cells can make the therapy done to the person much more effective.
 * They are also trying to treat or change cells in the immune system so that they can be strong enough to fight against the AIDS virus and be able to kill it.
 * Scientists work with body cells that are called somatic cell gene therapy.
 * There is another type of gene therapy called germine gene therapy that concentrates on inserting genes on reproductive cells. The genes added to the person, would pass on to their offspring. However, people are worried about this type of therapy because it can easily alter the genotype of the offspring.
 * //In this photo you can see how scientists work with somatic cells (body cells).//**

[|World Book- Gene Therapy]

[|Somatic Cell Procedure]

Here is a video which will make you understand better the basics of Gene Therapy:

media type="youtube" key="imL1Zmi3mWk?fs=1" height="385" width="640"

http://www.youtube.com/watch?v=imL1Zmi3mWk

Advantages and Disadvantages of Gene Therapy
__Advantage:__ When it works, it can help a person avoid a disease that they otherwise would have, and help them get away from suffering. (1, 2  __ Disadvantage: __ It would not necessarily work always, and when it would not, it could cause more problems, like cancer. (3 **__ Ethical: __** __ Advantage: __ It could protect babies from diseases, helping them to live a safer and longer life. (1, 2  __ Disadvantage: __ It could eventually lead to people choosing the traits that a baby would have. This would be considered unethical because it would be changing the way that the baby was meant to be. It could cause problems with equality to occur in the future. (4 To find results there have been and could be experiments done on humans. If failed, they could lead to further pain for a person, and possibly to death. (3, 5 **__ Economic: __**  __ Advantage: __ It is considered a cheaper way to cure genetic disorders, and would not be needed to be done as much as other methods may. (5 __ Disadvantage: __ It costs a lot to undergo gene therapy. (6 An example of this is: as of 1999 the cost could reach $100,000. (7
 * __Social:__**

1. [|“Gene Therapy”. Human Genome Project Information. 2010-11-16.]
2. [|Roan, Shari. “Gene Therapy May Reverse Down Syndrome”. 2010-11-16.] 3. [|Blaese, R. Michael. "Gene therapy." //World Book Student.// World Book, 2010. Web. 2010-11-16.] 4. [|Tucker, Patrick. “Genetically Designing Babies Is Ethical Under Certain Circumstances.” 2010-11-16.] 5. [|“Gene Therapy.” Genetic Engineering Debates. 2010-11-16.] 6. [|Danzon, Patricia, PhD; Towse, Adrian, MA. “The Economics of Gene Therapy and Pharmacogenetics.” 2010-11-17.]

7. [|Lane, Alfred, M.D. “Healing The Wounds that Never Heal.” Santa Clara University. 2010-11-17.]
Examples of gene therapy:

==== Assume that the rapidly dividing cancer cells form brain tumor. Some mutated or defective genes are the reason that brain tumor is forming. The gene therapy method used in this case would be inserting virulence removed herpes virus into the defective cells. The herpes virus that has its virulence removed is harmless,but its genetic materials are still capable to be inserted into the target cells. The herpes virus is first injected into mouse cells to make extra copies of itself. Then the virus containing mouse cells are injected into the tumor containing brain, the virus will find out the target tumor cells and attack them. After the insertion of the virus's genetic material, the tumor begins to produce herpes enzyme.Now the patient can be simply treated with herpes curing drug which will damage the tumor cell and the herpes producing mouse cells. )1 Scientists had this idea science 1972, they found that bacteria carries their genes in the small circular loops of additional DNA chromosomes. And only the germ cells have the genes that can be passed to the next generation. The manipulation of gene therapy started in the year of 1990.(2 ==== Bone marrow, skin, and liver cells. (3 The first human who accepted gene therapy treatment was a four-year old girl, Ashanti DeSilva. She was born with severe combined immune deficiency (SCID). SCID is a very rare genetic disease, it makes people lack of healthy immune system and defenseless to every passing germ. usually children with this disease couldn't survive to adult age, they die of overwhelming infections. Ashanti inherited a broken gene from both of her parents, so the instruction that the gene carries about manufactory protein called adenoside deaminase (ADA) is not functional. <span style="font-family: arial,helvetica,sans-serif; font-size: 13px; line-height: 19px;">Ashanti lived in a sterile environment at home and avoid any contacts with people other than her family and fight with illness frequently with large amounts of antibiotics. （4
 * ====Process of curing brain tumor====
 * <span style="font-family: Calibri,helvetica,sans-serif; font-size: 13px; line-height: 19px;">Other target that retroviruses is used
 * <span style="font-family: Times,helvetica,sans-serif; font-size: medium; line-height: normal;">The first human genetic therapy case

At the age of 4, Anshanti's parents found Dr French Aderson who had searched opportunity to perform gene therapy for long. The most effective treatment is blood cell transplantation. It has no risks since the blood cells which will be injected into Anshanti's body are her own blood cells. The doctors first took some of the blood cells from Anshanti's body, and let the cells grow in the lab. Then they inserted a hollowed-out virus which contains copies of ADA gene into the blood cells<span style="font-family: Times,helvetica,sans-serif; font-size: 16px; line-height: normal;">. <span style="font-family: arial,helvetica,sans-serif; font-size: 13px; line-height: 19px;">Finally, the blood cells were injected back into Anshita's body.（4

The gene therapy worked rapidly. Ashanti's T-cell (white blood cells) count rose to normal level, and her health kept improving during the next two years which allowed her to attend school. But Ashanti was not entirely cured, she still had to take a low dose of PEG-ADA to remain the function of white blood cells. （4

<span style="font-family: arial,helvetica,sans-serif; font-size: 13px; line-height: 19px;">1)process of curing brain tumor 2)[|principles and history] 3)[|other targets] 4）[|treatment process] [|treatment process]