Monday, March 1, 2010

Gene Therapy for ALD

We have been watching the movie Lorenzo's Oil in class the last few days.  Lorenzo has ALD. 

What is ALD?  What does it do to a person's nerve cells?  How does someone get ALD?

 Scientists now may have a way to treat boys with ALD using gene therapy.  Read the following article:  Gene Therapy Technique Slows Brain Disease ALD Featured In Movie 'Lorenzo's Oil'

What is gene therarpy?  How is the HIV virus used? 

21 comments:

  1. cherrell 5..

    Gene therapy is when an defective gene is replaced by a healthy gene.gene therapy can treat ALD. ALD stands for Adrenoleukodystroph
    which is basically a type of brain damage.It strippes the brains nerves cells. ALD is inheirted by The disorder is called X-linked because the mutated gene is on the X chromosome, inherited from the mother.

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  2. ALD is inherited from the mother and only affects boys. The disease start affecting a boy at 6-8 years old. The disease affects the myelin sheath that surronds brain nerves. The myelin sheath protects the brain nerves. The disease slowly tears apart this protective layer and the person slowly loses all of their mental and physical abilities. The gene therapy they are researching uses vectors based on the HIV virus to put corrected healthy ALD DNA into the patient.Once the healthy DNA is in the person the scientists they kill the old bone marrow. So far the result have been good and delayed the disease.

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  3. With ALD your brain can not work normaly which means that you will not be a noramal person for the rest of your life. However if you have this diseas then you will not be able to live your teenage life because you will probably die. This dieseas is more common on boys than girls. As time goes on the diseas gets more worse. This is really sad that it happens to young boys that havent actually lived their life. Can there be a cure for this.

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  4. ALD is a disease in which the body cannot digest certain saturated fats. These fats eventually affect the body’s neurons, which affect movement. The neurons and fibers in the brain are covered in a protein coat of myelin. When the myelin coat starts to deteriorate, the body cannot react to stimuli. ALD is an X- linked trait that only affects males. Scientists have now discovered an HIV- derived lentivirus vector that could help cure ALD. Lentivirus vectors come from a family of retroviruses, meaning that they will be in the body for life. Gene therapy is a process where a gene can be inserted onto another chromosome. If this particular vector is inserted onto a chromosome, it will be in the body forever, picking up the slack that the enzymes have lost in metabolism because of ALD.

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  5. ALD is a hereditary disease (meaning that is an X linked trait being passed down by the mother) in which there is loss of many fatty acids. The disease deteriorates the myelin sheath around nerve fibers in the brain that help the nerves to function properly. In gene therapy, a defective gene is replaces with a working one. HIV is being used in ALD gene therapy because retroviruses such as HIV have to be inserted into the chromosomes. This means that when cells with this gene divide, they will have the therapeutic gene forever and wont have to go in for regular treatment.

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  6. ALD is when the myelin sheath is starting to like evaporate you could say.It just doesn't protect the brain nerve cells anymore.The brains nerve cells start to deteriorate and the person would loose their physical and mental abilities. This starts to take place around 6 to eight years old.ALD is passed from the child's mother.Thee is no possible way that it could be passed by the father.

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  7. ALD is a genetic disease where saturated fats cannot be digested. These fats linger in the blood stream and starts to remove the myelin from the axons of the brain. This causes the axons not to transmit impulses, and the brain slowly begins to disintegrate. Eventually the disease gets to the point where the brain stops functioning, and you die. Because all boys who get the disease die, no girl can be ill with ALD because the disease is an X recessive, and a girl has 2 X's. They can be a carrier of the disease, and all boys who have it get it from their mother.

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  8. ALD is a progressive degenerative myelin disorder meaning that the insulation around the nerves slowly breaks down. This disease can only get worse over time since your body is unable to produce replacement myelin. You can get ALD through genetics. A mother can pass the gene to the children, the male offspring develops the disease while the women are just the carriers.

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  9. in the m ovie lorenzos oil, lorenzo had the symptoms of random tantrums, bleeding through the mouth and not being able to hear vry well. They soon diagnosed him with ALD which only affects males but is carried through the mother. ALD is when your body doesnt have enough myelin sheath or fatty acids and u have an abnormally high level of saturated fats i your blood and the long chains are unable to burn off. Your nerve cells become useless and you begin to lose your senses. Now that they have a cure for ALD boys (lorenzos oil) they have saved many lives.

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  10. ALD causes the saturated fats in a person's body to go undigested, and these lingering fats linger in the blood stream and reomove the myelin from the axons in the brain. The loss of the myelin causes the axons not to transmit impulses to the rest of the body, and the brain begins to slowly disintegrate. The disease finally gets to the point where the brain stops working correctly, and death soon comes. The disease is on the X-chromosome and is passed from mother to child, but ALD is only active in males and the females are just carriers.

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  11. ALD is a disease that is genetically passed from mother to child. It causes the myelin around the nerve cells of a human, which causes a number of symptoms, primarily from the inability of messages from the brain to parts of the body. French researchers have developed a special strain of HIV that can treat such diseases as ALD. This technique is a form of gene therapy, which has been designed to treat patients with such diseases to prevent ALD symptoms in infected children.

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  12. People that have ALD slowly lose their myelin sheath which a layer that protects the nerve fibers in the brain. Without this layer, the brain is not able to correctly function, which creates several didibilities. If a person has ALD, they can cure it by taking Lorenzo's Oil. Only boys can get this disease because it is a X linked. A person can get ALD through birth, a mother can carry it but she does not have it. So when she has a baby, the child, if a boy, can be born with ALD.

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  13. ALD is a genetic disorder where the body is unable to break down fatty acids and the myelin sheath slowly goes away. When the myelin is lost then the nerves start to lose their way of functioning. A person can get ALD from their mother and the disease is most common in young boys, however it is now possible for girls to get it as well. Gene therapy is when genetic engineering is used to try to cure a disease which has resulted due to a defect (genetic). The HIV virus is used because lentiviral vectors are used (which come from disabled HIV)in a variety of diseases.

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  14. ALD is a hereditery condition that results in loss of myelin sheath, which leads to increasing medical and physical disabilities. Gene therapy is the insertion of genes into an individual's cells in order to treat a disease, mostly hereditary. The HIV virus could possibly be used to treat many diseases.

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  15. ALD stands for Adrenoleukodystroph. ALD is inherited from the mother and passed onto the child, also ALD taking over. ALD is when the body cannot digest certain saturated fats and only affects boys. When the boy is about 6 to 8 years old, ALD will start. These fats affect the body’s neurons (which affect movement). The neurons and fibers in the brain are covered in a protein coat of myelin, the myelin coat starts to disappear, then the body cannot react to stimuli. The brain slowly begins to disintegrate, and stops functioning eventually leading to death.

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  16. ALD is sex-linked recessive gene that is only passed from mother to son. It is a very rare disease. If you have ALD your body cannot digest saturated fats and consuming these cause the myelin sheath in your neurons to dissolve causing many harmful and deadly symptoms. Some symptoms include: becoming blind and death, having seizures, and other painful reactions. Gene therapy can help people that have ALD by inserting genes that will help defeat the disease into their cells.

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  17. ALD is inherited from the mother and only affects boys. The disease start affecting a boy at 6-8 years old. The disease affects the myelin sheath that surronds brain nerves. The myelin sheath protects the brain nerves. The disease slowly tears apart this protective layer and the person slowly loses all of their mental and physical abilities. Gene therapy will help patients that have ALD to change the genes that the disease have and it changes its chromosome and that's how scientist think they can "cure" ALD

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  18. ALD is a disease in which the body can't digest specific saturated fats. These fats eventually affect the body’s neurons, which affect movement. ALD is passed from mother to child but only effects boys. Symptoms do not occur until the child is between 6-8 years of age.Scientists discovered that gene therapy can slow the process of ALD which can kill those who are infected usually before they reach adalescents. Gene therapy is where cells and removed from the body and modified then replaced back into the person. In the case of ALD the cell must be altered so that it can create a myelin sheath.

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  19. ALD is a disease similar to ALS except that you get ALD from your birth mother if she had inherated from her mother as well. The difference is that only boys are the ones that take the effects of the disease. You loose your ability to see, hear, talk, move, and swallow. You usually get twenty four months with the disease and then you die. The disease takes off the myelin sheaths from the neurons, so you loose the ability to control your body.Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease.

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  20. The progression of the genetic disease ALD is typically slowed by bone marrow transplants. These take a long time to find a match and are a risky procedure. A new possibility for reversing damage is gene therapy. Working, ALD-free genes are carried by lentivirus (HIV-like) vectors to the patients cells. In some tests, patients have shown improvement with this treatment, though it is also risky and could disturb cell biology (because of the virus DNA). Still, it has proven more effective than bone marrow transplants and may possibly be used as an ALD treatment in the future.

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  21. Adrenoleukodystrophy or ALD is a hereditary disease that travels on the X chromosome. It mostly effects boys. This disease, as seen in Lorenzo's Oil, destroys the myelin sheath of the nerves in an infected body.The myelin protects the brain's nerve cells, without it patients suffer severe brain damage. Most patients die before their tenth birthdays. Although Lorenzo's oil has seemed to be a cure, for patients that already have ALD, the brain damage is irreversible.

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