1. Gene Therapy

2. What is Gene Therapy? Gene Therapy is the insertion of genes into an individual’s cells and tissues to treat a disease. Gene Therapy is the insertion of genes into an individual’s cells and tissues to treat a disease. This technology is still being experimented with today, but it has been used with some success. This technology is still being experimented with today, but it has been used with some success.

3. How Does Gene Therapy Work? There are many different ways of correcting faulty genes: There are many different ways of correcting faulty genes: - A normal gene is inserted into a nonspecific location in the genome to replace a nonfunctional gene, this is the most common method. - An abnormal gene could be swapped for a normal gene through homologous recombination - The abnormal gene could be repaired through selective reverse mutation, which returns the gene to its normal function - The regulation of a particular gene( whether a gene is turned on or off) could be altered

4. How Does Gene Therapy Work? (continued) The Basic Process: A gene that is inserted directly into the cell usually will not function. Carrier molecules called vectors are genetically engineered to deliver the genes. Viruses are often used as vectors because they can deliver the new gene by infecting the cell. The viruses are modified so they do not cause disease when used in humans. A gene that is inserted directly into the cell usually will not function. Carrier molecules called vectors are genetically engineered to deliver the genes. Viruses are often used as vectors because they can deliver the new gene by infecting the cell. The viruses are modified so they do not cause disease when used in humans. A vector can be injected or given intravenously, directly into a specific tissue in the body, where it is taken up by individual cells. Also, a sample of the patient’s cells can be removed and exposed to the vector in a laboratory. The cells containing the vector are then returned to the patient. If the treatment is successful the new gene delivered by the vector will make a functioning protein. A vector can be injected or given intravenously, directly into a specific tissue in the body, where it is taken up by individual cells. Also, a sample of the patient’s cells can be removed and exposed to the vector in a laboratory. The cells containing the vector are then returned to the patient. If the treatment is successful the new gene delivered by the vector will make a functioning protein.

5. Diagram of Gene Therapy

6. The Applications of Gene Therapy When genes have been safely transferred and are being expressed their activity typically falls off after a short time. Because of this, trials now under way in humans are not aimed at correcting genetic defects; instead researchers are looking for ways to use gene therapy to counter major killers such as heart disease and cancer. In these cases a short period of activity by the transferred gene is not only sufficient but desirable. One application is to help treat coronary heart disease by introducing a gene encoding a growth factor that stimulates new blood vessels to grow around blocked arteries into the heart muscle. The goal is to get the heart cells to produce enough growth factor to trigger a brief period of blood vessel growth.

7. The Heart

8. The Benefits of Gene Therapy - Gene therapy may someday be used to treat a variety of diseases - Gene therapy can provide ‘patient-friendly’ treatment regimens for a variety of diseases. Today many patients with hemophilia and diabetes must have repeated injections in order to manage their disease because proteins exist in the blood stream for a limited amount of time before they are degraded. Since DNA is more stable and functions inside the cell, the delivery of genes may result in longer-term expression of the proteins. - Because gene therapy is accurate it has the potential to eliminate cancer cells without damaging normal, healthy tissue. - Gene therapies may provide alternatives when a disease does not respond to other treatments.

9. Ethical Issues What is considered normal and what is considered a disability or disorder, and who decides? What is considered normal and what is considered a disability or disorder, and who decides? Are disabilities diseases? Do they need to be cured or prevented? Are disabilities diseases? Do they need to be cured or prevented? Does searching for a cure demean the lives of individuals currently affected by disabilities? Does searching for a cure demean the lives of individuals currently affected by disabilities? Preliminary attempts at gene therapy are extremely expensive. Who will have access to these therapies? Who will pay for their use? Preliminary attempts at gene therapy are extremely expensive. Who will have access to these therapies? Who will pay for their use? Gene therapy may someday have the potential of allowing parents to choose the genes their children receive, is this ethical? Gene therapy may someday have the potential of allowing parents to choose the genes their children receive, is this ethical?

10. Bibliography http://images.google.ca/imgres?imgurl=http://www.wellesley.edu/Biology/Courses/219/Gen_ news/i3_Gene_Therapy.jpg&imgrefurl=http://www.wellesley.edu/Biology/Courses/219/Gen_ news/gen_news.html&h=441&w=591&sz=61&hl=en&start=42&tbnid=tcY0fOPpGFN5AM:& tbnh=101&tbnw=135&prev=/images%3Fq%3Dgene%2Btherapy%26start%3D40%26gbv%3D 2%26ndsp%3D20%26svnum%3D10%26hl%3Den%26safe%3Dactive%26sa%3DN http://images.google.ca/imgres?imgurl=http://www.wellesley.edu/Biology/Courses/219/Gen_ news/i3_Gene_Therapy.jpg&imgrefurl=http://www.wellesley.edu/Biology/Courses/219/Gen_ news/gen_news.html&h=441&w=591&sz=61&hl=en&start=42&tbnid=tcY0fOPpGFN5AM:& tbnh=101&tbnw=135&prev=/images%3Fq%3Dgene%2Btherapy%26start%3D40%26gbv%3D 2%26ndsp%3D20%26svnum%3D10%26hl%3Den%26safe%3Dactive%26sa%3DN http://en.wikipedia.org/wiki/Gene_therapy http://en.wikipedia.org/wiki/Gene_therapyhttp://en.wikipedia.org/wiki/Gene_therapy http://www.ornl.gov/sci/techresources/Human_Genome/medicine/genetherapy.shtml http://healthcare.utah.edu/healthinfo/adult/cardiac/images/arteries.gif http://www.ornl.gov/sci/techresources/Human_Genome/medicine/genetherapy.shtml http://healthcare.utah.edu/healthinfo/adult/cardiac/images/arteries.gifhttp://www.ornl.gov/sci/techresources/Human_Genome/medicine/genetherapy.shtml http://healthcare.utah.edu/healthinfo/adult/cardiac/images/arteries.gifhttp://www.ornl.gov/sci/techresources/Human_Genome/medicine/genetherapy.shtml http://healthcare.utah.edu/healthinfo/adult/cardiac/images/arteries.gif http://www.kfdunn.com/vector/gene-therapy.gif http://www.kfdunn.com/vector/gene-therapy.gifhttp://www.kfdunn.com/vector/gene-therapy.gif Biology Concepts and Connections, Fourth Edition, AP Biology Textbook Biology Concepts and Connections, Fourth Edition, AP Biology Textbook