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Gene Therapy - Involves delivery of therapeutic genes into the human body to correct disease conditions created by faulty

genes. Some of these diseases caused by faulty genes are: a) Cystic Fibrosis - passed down through families, causes thick, sticky mucus to build up in the lungs, digestive tract, and other areas of the body b) Huntingtons disease - an inherited genetic disorder that affects the brain and eventually mental and motor function and control c) Hemophilia a bleeding disorder in which it takes a long time for the blood to clot d) Sickle cell disease - is a serious disorder in which the body makes sickle-shaped red blood cells. Two Primary Gene Therapy Strategies: 1. Ex Vivo Gene Therapy a. Cells from a diseased person are removed b. They are then treated in the laboratory (e.g. bacterial transformation) c. Finally, they are reintroduced to the patient *Bacterial transformation - introduces a plasmid (small circular piece of DNA about 2,000 to 10,000 base pairs that contains important genetic information for the growth of bacteria) into a bacteria to make large quantities of itself * Transfection - the introduction of DNA into animal or plant cells 2. In Vivo Gene Therapy Introducing genes directly into tissues or organs without removing body cells The challenge is the delivery only to intended tissues Viruses act as vectors for gene delivery, but some injected directly into tissue Some of the Different Types of Viruses Used as Gene Therapy Vectors:

Retroviruses - A class of viruses that can create double-stranded DNA copies of their RNA genomes. These copies of its genome can be integrated into the chromosomes of host cells. Human immunodeficiency virus (HIV) is a retrovirus. Adenoviruses - A class of viruses with double-stranded DNA genomes that cause respiratory, intestinal, and eye infections in humans. The virus that causes the common cold is an adenovirus. Adeno-associated viruses - A class of small, single-stranded DNA viruses that can insert their genetic material at a specific site on chromosome 19. Herpes simplex viruses - A class of double-stranded DNA viruses that infect a particular cell type, neurons. Herpes simplex virus type 1 is a common human pathogen that causes cold sores.

* Since viruses are extremely effective at infecting human cells, they must be carefully engineered so that they can neither spread nor produce disease.

*Dr. Richard Mulligan of MIT (Massachusetts Institute of Technology) has synthetically created the perfect retrovirus: it has no reproduction sequence and exists solely to deliver therapeutic DNA during gene therapy. It has no viral DNA (DNA that would make the cell--and you-- sick) whatsoever and only carries the new DNA that has been spliced into it. Aside from Ex Vivo and In Vivo, we also have the Gene Silencing Method. Gene Silencing Also called antisense technology. With this method, geneticists can inactivate a gene that may cause disease or be defective. When DNA replicates, RNA bonds to half of the split double helix, making a mold of sorts. The RNA (messenger RNA or mRNA) is then used to create an identical DNA strand. To silence a gene on a chromosome, scientists, therefore, simply make an RNA strand 15-20 bases in length complementary to the mRNA. The synthesized RNA will attach itself to the mRNA and prevent that portion of the mRNA from creating the gene on the duplicate DNA strand.. Gene silencing is used to treat several viruses including AIDS, Herpes, Chicken Pox, and Hepatitis. More importantly, though, antisense technology is used by geneticists in research to learn what happens when certain genes are silenced.

What factors have kept gene therapy from becoming an effective treatment for genetic disease?

Short-lived nature of gene therapy - Before gene therapy can become a permanent cure for any condition, the therapeutic DNA introduced into target cells must remain functional and the cells containing the therapeutic DNA must be long-lived and stable. Problems with integrating therapeutic DNA into the genome and the rapidly dividing nature of many cells prevent gene therapy from achieving any long-term benefits. Patients will have to undergo multiple rounds of gene therapy. Immune response - Anytime a foreign object is introduced into human tissues, the immune system is designed to attack the invader. The risk of stimulating the immune system in a way that reduces gene therapy effectiveness is always a potential risk. Furthermore, the immune system's enhanced response to invaders it has seen before makes it difficult for gene therapy to be repeated in patients. Problems with viral vectors - Viruses, while the carrier of choice in most gene therapy studies, present a variety of potential problems to the patient --toxicity, immune and inflammatory responses, and gene control and targeting issues. In addition, there is always the fear that the viral vector, once inside the patient, may recover its ability to cause disease. Multigene disorders - Conditions or disorders that arise from mutations in a single gene are the best candidates for gene therapy. Unfortunately, some the most commonly occurring disorders, such as heart disease, high blood pressure, Alzheimer's disease, arthritis, and diabetes, are caused by the combined effects of variations in many genes. Multigene or multifactorial disorders such as these would be especially difficult to treat effectively using gene therapy.

Other information: First human gene therapy - Ashanti de Silva (4 years old) with severe combined immunodeficiency (SCID) treated in 1990 at NIH in Maryland

2011 Six patients with hemophilia were given a virus that infects the body with the blueprints needed to produce blood-clotting proteins. Four of them could then stop taking their drugs. Filipinos Former President Joseph Estrada has also undergone the stem cell therapy last May this year. Estrada said he received 14 shots of blood from unborn sheep in his buttocks during the visit. He said that it was a one-time thing and there will be no follow-ups. Estrada was told it would take three months before he can experience the full effects of the procedure. On the same month, 88-year-old Senate President admitted that he has also undergone the stem cell treatment procedure just like Former President Joseph Estrada.

References: www.mcvsd.org/weblog/dfusco/5GeneTherapy.ppt http://www.ncbi.nlm.nih.gov/pubmedhealth/PMH0001167/ http://www.medicinenet.com/huntington_disease/article.htm http://www.ncbi.nlm.nih.gov/pubmedhealth/PMH0001564/ http://www.nhlbi.nih.gov/health/health-topics/topics/sca/ http://www.ornl.gov/sci/techresources/Human_Genome/medicine/genetherapy.shtml http://library.thinkquest.org/28599/gene_therapy.htm http://www.newscientist.com/article/dn18819-gene-silencing-prevents-its-first-human-disease.html http://www.newscientist.com/article/dn10203-rna-interference-scoops-nobel-prize-for-medicine.html http://www.bbc.co.uk/news/health-16107411 http://www.bbc.co.uk/news/health-11313273 http://newsinfo.inquirer.net/192391/joseph-estrada-defies-age-shares-how-he-did-it-stem-cell-therapy http://www.philstar.com/Article.aspx?publicationSubCategoryId=63&articleId=812538